January 2023 Newsletter

Jill Beirl

Message from the Executive Director  |  Rare Disease Week Advocacy Stipend​  |  Community Meeting with Azafaros  |  Rare & Ready Advocacy Bootcamp​  |  Courageous Parents Network Virtual Workshop​  |  Thanking our Niemann-Pick Health Care Heroes​  |  ASMD Survey Opportunity  |  Milestones​  |  Bringing Holiday Cheer Update​  |  AmazonSmile Update  |  The Assistance Fund  |  Surveys, Studies, & Market Research  |  Clinical Trial Updates​  |  Community News Updates​  |  Fundraising  |  NNPDF Membership  |  Emergency Hardship Program

Message from the Executive Director

Dear NNPDF Community,

It is hard to believe that we are already a month into the new year. With Rare Disease Day just a few weeks away, this is a great opportunity to express our gratitude to the many members of the Niemann-Pick disease community who support our work at NNPDF. We would also like to take this opportunity to thank you for your continued support and to highlight the many ways that these efforts are making a positive difference.

On February 28th, patients, families, friends, clinicians, researchers, industry leaders and policy makers will join together to raise awareness of the issues impacting more than 300 million people around the world who are affected by rare diseases. At NNPDF, we will be launching a range of initiatives to help raise awareness of the impact of Niemann-Pick disease on social media, through blog posts and LinkedIn articles, and by continuing our efforts to highlight the critical need for research that can lead to new treatments.

This year’s Rare Disease Day theme is “Share Your Colors.” This highlights the importance of elevating all our voices to make sure our needs are being heard by people who can help make a difference. By using your voice to share your story, you can help more people learn about Niemann-Pick disease. By sharing your story, you can help more people get the screening they need for a proper diagnosis as early as possible and perhaps most importantly, your story can help more families learn that they are not alone and that there is support available for the emotional, financial, or medical issues they might be facing. Raising awareness also helps leaders in government, industry, and research to better understand our needs and develop policies or research studies that can lead to better support and more hope for patients and families in the years ahead. Your story can also help shine light on our mission at NNPDF and help more people to connect with our information and services.

As we plan make the most of Rare Disease Day, we also emphasize that NNPDF is dedicated to raising awareness every day to make sure our community has the support it needs. We continue to expand our support services to bring more resources to both patients and caregivers each year. We are in contact with regulators and industry leaders to ensure we bring them new insights on ways to support our community. We will also support new research that can highlight all of the important issues that affect patients and caregivers. And as always, we continue to fight for research that can lead to new treatments and will not stop until ALL people impacted by ALL types of Niemann-Pick disease have access to the therapies they need.

We hope you will join us in our efforts to raise awareness in 2023 and send our best wishes to you and your family at the start of this new year.

Sincerely,
Joslyn Crowe,
NNPDF Executive Director

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Rare Disease Week on Capitol Hill Advocacy Stipend

Rare Disease Week on Capitol Hill events will be taking place from February 28 – March 2, 2023 in Washington, DC. NNPDF is offering a limited number of NNPDF Advocacy Stipends for Niemann-Pick individuals, caregivers, or family members to attend! Complete details can be found here.

Rare Disease Week on Capitol Hill brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. 

NNPDF Advocacy Stipend information and registration

Visit everylifefoundation.org for Rare Disease Week on Capitol Hill information on:

Now more than ever, it’s important for Niemann-Pick families to communicate our priorities and concerns to decision-makers and leaders!

Community Meeting with Azafaros

NNPDF and NTSAD invite you to join us for a Community Meeting with Azafaros to learn more about their Phase 2 multi-national trial for AZ-3102 in Niemann-Pick Disease Type C and GM2 Gangliosidosis on Wednesday, February 8th at 8:00pm ET. Speakers include Daniel Mitroi, MD, Regional Medical Director, Azafaros.

Register to attend.

AZ-3102 is a therapeutic candidate developed for people affected by Lysosomal Storage Disorders (LSDs) with neurological involvement. Azafaros B.V. recently received Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) earlier this month, to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, which is being investigated for the treatment of neurological symptoms of Niemann-Pick disease type C (NP-C) and GM2 gangliosidosis (GM2).

Rare & Ready Advocacy Bootcamp

Please join Rare & Ready: A Genetic Condition Coalition for a 90-minute Advocacy Bootcamp on Tuesday, February 7, at 12:30 pm ET.  All diagnosed individuals and family members are invited to attend this free webinar!

Jennifer Hitchon and Ken Sprague, Government Affairs at BioMarin, will share information on state policies important to the rare disease community.

You will learn:

  • How to communicate with your state legislator
  • How to use social media effectively
  • Advocacy strategies to help you navigate policy hurdles

NNPDF is a member of the Rare & Ready Coalition.

Pre-registration is required. Learn more.

Courageous Parents Network Virtual Workshop

Courageous Parents Network invites you to join their upcoming virtual workshop: Managing Caregiving Stress as a Couple on Wednesday, February 15th at 8:00 pm ET

All relationships face stress, but partners caring for a child with a serious illness are confronted with unique challenges. Dr. Talia Zaider, a licensed psychologist and marriage and family therapist, will offer insights and considerations for coping together. Register today!

The workshop is free and we hope you will take advantage of this offering.

Thanking our Niemann-Pick Health Care Heroes

Clinical care team members play an important role in the lives of our Niemann-Pick families and are valued throughout our community. In recognition of Rare Disease Day NNPDF will mail Thank You cards to your Niemann-Pick Health Care Heroes to let them know they are very important to us. Health Care Heroes can include your primary care provider, genetic counselor, speech pathologist, physical or occupational therapist, gastroenterologist, respiratory therapist, home health care nurse, etc.

Participate by providing us your Health Care Heroes’ information by February 20th.

Coming Soon...

Acid Sphingomyelinase Deficiency (ASMD) Patient Experience with Olipudase Alfa Survey

The International Niemann-Pick Disease Registry (INPDR) is conducting this survey in collaboration with The National Niemann-Pick Disease Foundation, Inc. (NNPDF), International Niemann-Pick Disease Alliance (INPDA), and Niemann-Pick UK (NPUK). This survey forms part of a larger study that includes interviews with Niemann-Pick disease type A/B and type B (ASMD) patients, which will explore the questions in this survey in more depth.

Survey information will be released shortly by eblast and on our website or contact [email protected] to participate.

Milestones

Do you have a special milestone to share? Send us a photo along with the details to [email protected] and we’ll publish in an upcoming newsletter!

Congratulations Tinley Apt who has been doing remarkable since starting enzyme replacement treatment for ASMD in August 2021. She was so excited to get to play soccer last summer and keeps busy and active with coloring, playing with friends and loves to swim in the summer.  GO TINLEY!!

Congratulations to Evren Ayik! Evren was selected by Uplifting Athletes as a Broncos Country Hero of the Game at the Sunday, January 8th game between the Denver Broncos and the Los Angeles Chargers. Evren shares “One of the best experiences of my life! Thank you to the Uplifting Athletes and Denver Broncos for this very special and memorable experience!” Way to go Evren!! You can check out the video on Facebook.

Bringing Holiday Cheer Update

Twenty-six NNPDF Community member families had help in making their holidays a little brighter. A heartfelt THANK YOU to our special anonymous donor. This special program aims to help ease the burden of holiday stress, while promoting family togetherness and enjoyment, by helping to bring some cheer to our Niemann-Pick families. NNPDF was able to assist with over 200 items for our families. Thank you again for providing much joy to our families.

AmazonSmile Ends February 20th

AmazonSmile has made an impact in our community by donating over $6000 to NNPDF since it started thanks to those of you who signed on to their giving program. Unfortunately we’ve been informed that the AmazonSmile program will be ending February 20th. Please continue to support this program until then. We like to thank all those that contributed to NNPDF through the AmazonSmile charity program.

The Assistance Fund

Struggling with out-of-pocket expenses due to acid sphingomyelinase deficiency (ASMD)? The Assistance Fund is an independent charitable patient assistance organization helping patients and families by providing financial assistance for the cost of treatment and other health-related services. Through its Acid Sphingomyelinase Deficiency Financial Assistance Program, eligible individuals can receive assistance for all FDA-approved treatment for ASMD, including:

  • Prescription drugs (copays, deductibles, and coinsurance)
  • Health insurance premiums
  • Therapy administration costs
  • Disease management (such as prescribing-physician copayments), treatment-related travel costs, and genetic testing

Learn more about The Financial Assistance program.

Surveys, Studies, & Market Research

Be sure to check out our Surveys & Market Research webpage for current survey and study opportunities in the Niemann-Pick disease space. Participating in surveys and studies is important to the advancement of health options for our Niemann-Pick community members. Contact Laurie Turner at [email protected] for any questions.

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

Updates from Azafaros:
Azafaros receives FDA IND clearance and Fast Track Designation for its lead asset AZ-3102 for treatment of Niemann-Pick disease type C (NPC). Read complete announcement.

Azafaros announces organizational changes within the company. Read press release.

Updates from Cyclo Therapeutics:
Cyclo Therapeutics’ January 2023 newsletter is live. Scott Fine, CEO shares an update on their ongoing Phase 3 study, TransportNPC™ and a recap of key activities delivered last year. Read more.

Cyclo Therapeutics provides clinical program update and highlights recent achievements. Read complete update.

Community News Updates

Updates from KemPharm:
KemPharm demonstrates its continuing commitment to the Niemann-Pick Community with recent leadership changes. Read press release.

KemPharm announces board and leadership changes to support its transformation into a leading rare disease company. Read complete announcement.

KemPharm announces the promotions of Sven Guenther, PhD, to Chief Scientific Officer and Christal Mickle, MA, to Chief Product Development Officer. Read press release.

Update from Cyclo Therapeutics:  Cyclo Therapeutics has announced its abstract has been accepted for poster presentation at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting regarding TransportNPC. Read the complete announcement.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters!

THANK YOU to Amanda Stephens, Kasey Apt, Rick Alan, Jessica Stull, Haley Coulter Wilson, Kathleen Margaret, and Kit Neal Hensler who recently hosted Facebook Fundraisers for the NNPDF! We truly appreciate your support!

Want to host your own Facebook Fundraiser? It’s easy! Visit facebook.com/fund/NNPDF to get started!

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Emergency Hardship Program

The NNPDF Emergency Hardship Program continues to offer assistance to qualified NNPDF U.S. member families facing a crisis. Funding includes but is not limited to, specialized medical equipment, durable medical goods, utility bills (heating and cooling, electricity, phone, water and sewer), home and car repairs, and bereavement expenses. Click here for complete details and eligibility information.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

            

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The Assistance Fund – ASMD Financial Assistance Program

Jill Beirl

The Assistance Fund’s ASMD financial assistance program provides financial assistance for the following:

  • Prescription drug assistance (copays, deductibles, and coinsurance) on all FDA-approved treatment for ASMD
  • Health insurance premiums
  • Therapy administration costs
  • Disease management (such as prescribing-physician copayments)
  • Treatment-related travel costs
  • Genetic testing

The Assistance Fund (TAF) helps patients and families facing high medical out-of-pocket costs by providing financial assistance for co-payments, coinsurance, deductibles, and other health-related expenses. Since its founding in 2009, TAF has helped more than 160,000 children and adults in all 50 states, Washington, DC, and Puerto Rico.

Among TAF’s 80 disease programs is the Acid Sphingomyelinase Deficiency (ASMD) Financial Assistance Program. The Acid Sphingomyelinase Deficiency Financial Assistance Program provides financial assistance for out-of-pocket costs associated with all FDA-approved treatment for ASMD, including prescription drugs (copays, deductibles, and coinsurance), health insurance premiums, therapy administration costs, disease management (such as prescribing physician copayments), treatment-related travel costs, and genetic testing.

To be eligible for assistance, patients must be U.S. citizens or permanent residents, meet certain income requirements, have a diagnosis of the disease named in the disease program, have government or private health insurance, and a prescription for an FDA-approved treatment for ASMD. Once a patient is enrolled in a disease program, their coverage lasts the entire calendar year and there is no cap on the amount of assistance in that calendar year.

To learn more, or to apply today, visit enroll.tafcares.org or call (855) 233-0505.

ASMD Financial Assistance Program – One Pager
The Assistance Fund Press Release 01/16/2023

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The Benefits of Having a Great Local Care Team

Jill Beirl

Most patients and families in the Niemann-Pick community are too familiar with the need to travel to access the specialized medical care they need. Among our many services at NNPDF, patients often turn to us to find hospitals and doctors that have specific experience in treating Niemann-Pick disorders. We maintain a list of Comprehensive Care Centers that can help patients and families have access to Niemann-Pick specialists.

Despite these efforts, in many cases patients do not live in areas where medical specialists with experience in Niemann-Pick disease are nearby. They must then depend on non-specialists for much of their routine and emergency medical care. In these cases, it is important for patients and caregivers to identify the best strategies and options to build a strong local care team. These efforts are essential to make sure that patients have access to the full range of care services they need. 

By taking the time to research and build a strong local care team, patients can access a range of benefits including convenience, faster access to care, less time off from work or school, reduced costs and the comfort of knowing that support is available close to home if there is an emergency.

The best local team can differ significantly for different patients based on their health status and needs.  At a minimum, every Niemann-Pick patient should have a dedicated local primary care physician (PCP) to provide support for routine health needs. If the PCP is not familiar with Niemann-Pick disease, the patient and caregiver together should work to provide information about the disease that can help inform and guide treatment. This can also help the PCP be better prepared to monitor patient progress and medications between visits with a specialist and provide referrals as needed.

In addition to the PCP, the local care team might include many other specialists who can play an important role in care, including:  

  • geneticists
  • neurologists
  • gastroenterologists
  • pulmonologists
  • physical therapists
  • speech therapists

Patients should talk to both their PCP and their Niemann-Pick specialist about the different other medical experts they might need as part of a care team. From there, a PCP might be best positioned to help identify specialists in the area who might be a good fit. Once again, it can be helpful to provide every member of a local care team with information about Niemann-Pick disease and the patient’s full medical history.  At NNPDF our Family Resources page offers information that can be easily shared with any member of a medical team.

In any community, you might start a search for a PCP or other specialists by asking friends and family members for a referral. If you need assistance finding a physician, there are also several helpful online websites including ZocDoc, MediFind, and Vitals that can help identify doctors in your area.

NNPDF also often works with patients and caregivers to help identify medical experts who are available to work with patients at every step of their Niemann-Pick journey. Our experts are available to provide guidance and access to resources that can help you find local physicians and materials and strategies that can help make sure that your team is able to provide you with the very best support possible. For more information or if you have any questions, please reach out to us at [email protected].

 

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November-December 2022 Newsletter

Jill Beirl

Message from the Executive Director  |  We Remember Them  |  The Benefits of Having a Great Local Care Team  |  Dangerous Blues by Stephen Policoff  |  Gift Ideas Supporting the NNPDF  |  AmazonSmile  |  NPC Survey Opportunity  |  ASMD Accelerate  |  NPC Sibling Study |  Clinical Trial Updates  |  Community News Updates  |  Fundraising  |  Surveys & Studies  | Membership  |  Emergency Hardship Program

Message from the Executive Director and Board Chair

Dear NNPDF Community,

It is hard to believe that we are just weeks away from the end of 2022. And once again this has been a monumental year with many important developments for our community. We are  very pleased to have this opportunity to wish you and your family all the best for the New Year and to reflect on some of the important events of 2022.

Heading into 2023, NNPDF will work to expand our efforts and build on the momentum in 2022 in many new ways. We will continue to support and participate in research initiatives and other programs that can help leaders in government, industry, and advocacy better understand the impact of Niemann-Pick disease. We will continue to work with regulators to encourage them to consider the essential new approaches in clinical research that can lead to more new treatments in the years ahead. We also remain committed to fighting for access to treatment for the entire community and will not stop until ALL people affected by ALL types of Niemann-Pick disease have access to life-changing therapies. As the Niemann-Pick landscape changes and treatments do become available, we will also expand support to help families manage issues related to insurance, working with their providers, and life planning.

End-of-year is also a great time to recognize the many examples of strength and determination from our community and thank all the people – healthcare leaders, patients, families, researchers, donors, and industry partners – who supported our work at NNPDF. As we look back on the year, we are so encouraged by the amazing commitment of so many members of our community. We might be small, but we are mighty. The last year has shown us what is possible when we stay strong and work together.

Thank you again for your continued support. We are confident that in 2023 we will break many more barriers and reach new levels of change for the community. The battle is not over, but there is more hope on the horizon than ever before.

From all of us at NNPDF, have a safe and happy holiday.

Sincerely,
Joslyn Crowe, NNPDF Executive Director
Becky McGuire, NNPDF Board Chair

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We Remember Them

As a part of our Niemann-Pick community, please join the NNPDF in keeping all of our families in our thoughts now and throughout the year.

The Benefits of Having a Great Local Care

Most patients and families in the Niemann-Pick community are too familiar with the need to travel to access the specialized medical care they need. Among our many services at NNPDF, patients often turn to us to find hospitals and doctors that have specific experience in treating Niemann-Pick disorders… Check out our latest blog post entitled The Benefits of Having a Great Local Care Team.

Dangerous Blues by Stephen Policoff

Congratulations to Stephen Policoff, Niemann-Pick community member, on his recently published book Dangerous Blues. Stephen has generously cited NNPDF as the organization to which he wishes to donate a percentage of the royalties. Steven’s daughter Anna lost her battle in 2015 of NPC-related causes. This novel is dedicated to Anna and to his wife Kate, who also passed away in 2012.

Gift Ideas Supporting the NNPDF

Looking for a gift idea? Check out our Books and Publications webpage. Here you’ll find books authored by Niemann-Pick community members who have expressed themselves in books or other publications. Also included are books by authors that help raise awareness of Niemann-Pick Disease.

Shop AmazonSmile for the Holidays​

When you shop AmazonSmile this holiday season, be sure to designate National Niemann-Pick Disease Foundation as your favorite charity! It’s an easy way to make an impact for Niemann-Pick families. Shop AmazonSmile and Amazon will donate to the NNPDF. It’s easy! Learn more and sign up. You shop. Amazon gives.

NPC Survey Opportunity

Quality of life experience in patients with Niemann-Pick disease Type C during the COVID-19 pandemic

Are you an adult who has been diagnosed with Niemann-Pick disease type C (NPC) or the parent/caregiver of someone who has been diagnosed with NPC? If so, we would like to invite you to take part in our research study looking at quality of life in those with NPC. Learn more about this study.

Research reminder from Wylder Nation and NNPDF

Wylder Nation and NNPDF are still enrolling for ASMD Accelerate! Every child’s story can make a difference for the future of ASMD care.

This study is an opportunity to contribute to ASMD research from home. Signing up with PicnicHealth takes 5 – 10 minutes – after that PicnicHealth’s team collects your child’s longitudinal medical records on your behalf, securely upload them to your PicnicHealth account, and then de-identifies these records for ASMD researchers.

Visit picnichealth.com/asmd-wylder-nation or email [email protected] with any questions.

NPC Sibling Study

NNDPF, APMRF, Firefly Fund, and NP Canada have chosen to partner with the International Niemann-Pick Disease Registry (INPDR) moving forward and to continue the NPC sibling study through the INPDR. The INPDR is the Niemann-Pick specific patient registry which is led and governed by the Niemann-Pick community. We are recommending that all NPC families who have shared their loved one’s data with AllStripes have their data transferred to INPDR. This is completely voluntary and each family’s decision is respected. We have worked with AllStripes and the INPDR to ensure that this is a user-friendly and fairly simple process for families.

For information on how to easily share you loved one’s records with the INPDR, or if you have any questions, please contact us at [email protected].

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

Update from IntraBio:  IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301). Read the complete announcement.

Update from IntraBio:  IntraBio Ltd. is pleased to share that IB1001-301 has enrolled over 70% of the target number of patients in the second month of recruitment. Recruitment is expected to be completed by December 2022. Read the complete announcement.

Community News Updates

Update from Mandos Health:  Mandos Health has shared the following November 2022 update with the NNPDF. Read the complete update.

Update from KemPharm:  KemPharm has shared the following news release with the NNPDF, reporting their third quarter 2022 results. Read news release.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters!

THANK YOU to Julie Patenaude, Kit Neal Hensler, Charlotte Gibier, Jeff Padden, Donna Norberg, Joyce Gurtatowski, JoAnn Williamson, and Anne OConnor-Smith who recently hosted Facebook Fundraisers for the NNPDF! We truly appreciate your support!

Want to host your own Facebook Fundraiser? It’s easy! Visit facebook.com/fund/NNPDF to get started!

Surveys, Studies, & Market Research

Be sure to check out our Surveys & Market Research webpage for current survey and study opportunities in the Niemann-Pick disease space. Participating in surveys and studies is important to the advancement of health options for our Niemann-Pick community members. Current surveys and studies are listed below. Contact Laurie Turner at [email protected] for any questions.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Emergency Hardship Program

The NNPDF Emergency Hardship Program continues to offer assistance to qualified NNPDF U.S. member families facing a crisis. Funding includes but is not limited to, specialized medical equipment, durable medical goods, utility bills (heating and cooling, electricity, phone, water and sewer), home and car repairs, and bereavement expenses. Click here for complete details and eligibility information.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

            

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October 2022 Newsletter

Jill Beirl

Niemann-Pick Awareness Month  |  NNPDF In Action  |  Celebrating #NiemannPickChampions  |  Global Niemann-Pick Disease Awareness Day  |  Bringing Holiday Cheer  |  In the Spotlight  |  ASMD Accelerate  |  Pfrieger’s Digest  |  Clinical Trial Updates  |  Fundraising  |  Surveys & Studies  | Membership   |  Emergency Hardship Program

Niemann-Pick Awareness Month

Thank you to every #NiemannPickChampion for helping us continue to raise awareness of Niemann-Pick disease this October!  Together, your voices amplify our efforts to change the course of this rare disease.  Thank you for supporting NNPDF!

Niemann-Pick Awareness Month
#NiemannPickChampion

NNPDF In Action

Joslyn Crowe, NNPDF Executive Director and Kari Lato, NNPDF Board of Directors at the 2022 NORD Rare Summit. NNPDF is a member of the Rare & Ready Coalition.

Joslyn Crowe, NNPDF Executive Director with Sanofi’s Eric Racine, Vice President & US Country Head, Public Affairs & Patient Advocacy at Sanofi, and Stephen Meunier, Head of US Public Affairs & Patient Advocacy for Rare Disease, at Sanofi’s Patient Advocacy Council in Washington DC.

Joslyn Crowe with the Sanofi team at NORD Rare Summit viewing their poster on Survival of Patients with Acid Sphingomyelinase Deficiency in the United States: A Retrospective Real-World Study.

Celebrating #NiemannPickChampions during Niemann-Pick Awareness Month

As we celebrate Niemann-Pick Awareness Month this October, the National Niemann-Pick Disease Foundation will use this important observation to highlight and recognize many of the significant accomplishments achieved by members of our community over the last year. We can take this opportunity to recognize so many of our “Niemann-Pick Champions,” including heroic efforts by patients, caregivers, researchers, clinicians, and advocates who have contributed to making a positive difference in many ways. Champions from our community have helped us make historic progress in research and efforts to build awareness. So many have joined in efforts to help others. Our Champions are reaffirming our commitment to fighting for more treatments and services to support ALL people impacted by ALL types of Niemann-Pick disease and are continuing the fight to make access to care possible for everyone living with Niemann Pick disease. Read complete article.

Global Niemann-Pick Disease Awareness Day: The International Fight Continues

By Joslyn Crowe

Each year on the 19th of October the National Niemann-Pick Disease Foundation joins with the global community of patients, parents, caregivers, clinicians, and researchers in recognizing Global Niemann-Pick Disease Awareness Day. This day represents an international effort to build broader awareness of the impact this disease has on patients and families and is an important opportunity for all of us to join forces and raise our voices to highlight the issues affecting our community in countries around the world. It is also a time to reflect on the progress and challenges of the past year and to think critically about how we can all work together to have an even bigger impact in the coming years. As we celebrate the progress made in the past year, we can also reinforce our commitment to ensuring that ALL people impacted by ALL types of Niemann-Pick in ALL parts of the world have access to treatments, services and programs that can make a positive impact on their lives. Read complete article.

Bringing Holiday Cheer

NNPDF is fortunate to share that an anonymous donor has offered to help make the holidays a little brighter for Niemann-Pick Families! Our donor family wishes to help ease the burden of Holiday stress while promoting family togetherness and enjoyment by helping to bring some cheer to your family by providing wish list items. All NNPDF members residing in the US are eligible to submit an application to be considered for funding for the Bringing Holiday Cheer program. Application deadline is Friday, November 18th at 5pm ET. Click here for complete details.

 

Travis Obermeyer

Dad to Austin, ASMD
NNPDF Board of Directors

Tell us a bit about yourself.
Our family lives in the beautiful mountains of the Blue Ridge in North Carolina. We enjoy walking at the park, watching sports and spending time together on the Blue Ridge Parkway. Our vacation of choice is camping, as we love the solitude of being in God’s nature.

When did you receive Austin’s diagnosis and what led to this?
Austin’s pediatrician first noticed there was something off during his 15-month checkup when his liver was enlarged. Susan and I took Austin to the hospital that night for a series of tests with initial thought of Leukemia. All these tests were negative. After 6 months of further blood tests, ultrasounds, liver biopsy, enzyme tests etc., it was initially determined that he had Gaucher disease. However, upon further examination it was later confirmed to be ASMD (type B).

How did you learn about NNPDF?
We first learned about NNPDF through our geneticist at the time. Our family was lucky that she had trained under Dr. Wasserstein in NY and was familiar with ASMD in many facets.

What led you to get involved with NNPDF as a family and recently, as a Board Member?
I believe that as a parent you are the best advocate for your family and children’s well-being. Naturally that progresses to getting involved as much as possible and help any way to find new treatments/cures for the disease. I am a parent first and I want to fight and have a voice for Austin and other boys/girls and men/women that cannot fight for themselves.

How has being an NNPDF member supported your family?
NNPDF has helped our family most by creating connections. Connections with pertinent and accurate information. Connections with providers of treatment. Connections with other families going through similar circumstances.

What advice would you share with newly diagnosed families?
Dive in and learn about the disease as much as possible. Ask questions, no question is a bad question, we have all been in the same situation you are in. Again, as I said before, as a family member, friend or diagnosed individual you are your own best advocate. At first you may be overwhelmed with the plethora of information. Focus on what is important to you and reach out to NNPDF and connect with other families.

What are your hopes for the future for your family and for the Niemann-Pick community?
Ultimately, I am hopeful that NNPDF will continue to be instrumental in fostering the discovery of new treatments/cures for ASMD and NPC.

Research reminder from Wylder Nation and NNPDF

Wylder Nation and NNPDF are still enrolling for ASMD Accelerate! Every child’s story can make a difference for the future of ASMD care.

This study is an opportunity to contribute to ASMD research from home. Signing up with PicnicHealth takes 5 – 10 minutes – after that PicnicHealth’s team collects your child’s longitudinal medical records on your behalf, securely upload them to your PicnicHealth account, and then de-identifies these records for ASMD researchers.

Visit picnichealth.com/asmd-wylder-nation or email [email protected] with any questions.

Pfrieger's Digest

NNPDF is pleased to share the latest edition of Pfrieger’s Digest, written by Frank Pfrieger, PhD of Niemann-Pick Selbsthiifegruppe (Germany). This publication provides a summary of research advances based on selected peer-reviewed publications in scientific journals. Read the latest issue.

Clinical Trial Updates

Clinical trials are currently in progress to study and develop treatments for ASMD and NPC. The NNPDF posts new information regarding clinical trial updates as soon as it is received. Visit our Clinical Trials web page for up to date information on all clinical trials.

Update from Cyclo Therapeutics:  Cyclo Therapeutics announces the publication of Phase 1 data for Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease type C1. Read complete update.

Update from IntraBio:  IntraBio Ltd. is pleased to confirm the Phase III pivotal trial with N-acetyl-L-leucine (IB1001-301) is active and recruiting in the United States at the Mayo Clinic, MN. Read the complete announcement.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters!

THANK YOU to Krissy Florkiewicz, KayLa Miller, Sherri Sykes Lewis, and Kevin Xie who recently hosted Facebook Fundraisers for the NNPDF! We truly appreciate your support!

Want to host your own Facebook Fundraiser? It’s easy! Visit facebook.com/fund/NNPDF to get started!

Surveys, Studies, & Market Research

Be sure to check out our Surveys & Market Research webpage for current survey and study opportunities in the Niemann-Pick disease space. Participating in surveys and studies is important to the advancement of health options for our Niemann-Pick community members. Current surveys and studies are listed below. Contact Laurie Turner at [email protected] for any questions.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Emergency Hardship Program

The NNPDF Emergency Hardship Program continues to offer assistance to qualified NNPDF U.S. member families facing a crisis. Funding includes but is not limited to, specialized medical equipment, durable medical goods, utility bills (heating and cooling, electricity, phone, water and sewer), home and car repairs, and bereavement expenses. Click here for complete details and eligibility information.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

            

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Global Niemann-Pick Disease Awareness Day: The International Fight Continues

Jill Beirl

By Joslyn Crowe

Each year on the 19th of October the National Niemann-Pick Disease Foundation joins with the global community of patients, parents, caregivers, clinicians, and researchers in recognizing Global Niemann-Pick Disease Awareness Day. This day represents an international effort to build broader awareness of the impact this disease has on patients and families and is an important opportunity for all of us to join forces and raise our voices to highlight the issues affecting our community in countries around the world. It is also a time to reflect on the progress and challenges of the past year and to think critically about how we can all work together to have an even bigger impact in the coming years. As we celebrate the progress made in the past year, we can also reinforce our commitment to ensuring that ALL people impacted by ALL types of Niemann-Pick in ALL parts of the world have access to treatments, services and programs that can make a positive impact on their lives.

At NNPDF, we are dedicated to working to expand access to the care and support services that can help diagnosed individuals and caregivers manage a wide range of emotional, financial, and medical challenges. We are also dedicated to raising broader awareness on a global scale so that more people will understand the experience of living with NPD and more diagnosed individuals will get the help they need. We regularly meet with leaders in government and industry to ensure that they understand the challenges that patients and caregivers face every day. We collaborate with partners to conduct research that sheds new light on the issues affecting patients and caregivers in our community. We also worked tirelessly to develop and support initiatives that can help expand access to treatment. This is also a year where we experienced some historic moments with the approvals of a first treatment for ASMD in the U.S., Europe, Japan, and Brazil. This landmark event is an example of what we can achieve for the global community when we work together.

Reflecting on our achievements this past year, including the approval of a drug to treat ASMD, it’s clear how critical collaboration is in our field. Our community’s successes often come from collective efforts and shared knowledge. In much the same way, we continually seek new partnerships that offer mutual benefits, just as B2Cs lista över casinon utan spelpaus provides a resourceful database for players, listing gaming platforms without certain restrictions. This approach to shared information exemplifies our commitment to working with partner advocacy organizations, pooling resources, and supporting initiatives like the International Niemann-Pick Disease Registry. Through these collaborations, we gather vital data that strengthens our advocacy and empowers patients worldwide.

The challenges and accomplishments of the past year demonstrate that our global community is stronger than ever. We have come a long way, but the fight is not over. Let us continue working together, reaching across borders, staying hopeful, and fighting for our community’s right for access to life-changing treatments. The battle continues.

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Celebrating #NiemannPickChampions during Niemann-Pick Awareness Month

Jill Beirl

As we celebrate Niemann-Pick Awareness Month this October, the National Niemann-Pick Disease Foundation will use this important observation to highlight and recognize many of the significant accomplishments achieved by members of our community over the last year. We can take this opportunity to recognize so many of our “Niemann-Pick Champions,” including heroic efforts by patients, caregivers, researchers, clinicians, and advocates who have contributed to making a positive difference in many ways. Champions from our community have helped us make historic progress in research and efforts to build awareness. So many have joined in efforts to help others. Our Champions are reaffirming our commitment to fighting for more treatments and services to support ALL people impacted by ALL types of Niemann-Pick disease and are continuing the fight to make access to care possible for everyone living with Niemann Pick disease.

Over the past year, we have been so grateful to the patients and caregivers who have participated in meetings, shared their voices in webinars, posted information on social media, and interacted with local media and community leaders to make sure the needs of our community are being heard. Our Champions also include many dedicated clinicians and researchers who have continued to move research forward to help our community find more paths that can lead to new treatments. Every person who has advocated for our community in even the smallest way is Niemann-Pick Champion and we thank you for your continued support.

One of the greatest moments our community experienced this past year was the historic approval of a first ever treatment for ASMD in the U.S., Europe, and Japan. This landmark event is the result of years of dedication by so many stakeholders in industry as well patients, families, and other members of advocacy communities, and it shows what is possible when we join together in a common goal. We should all see this milestone not as an end, but as a beginning and a chance for renewed hope. All of our Champions know that there are many other members of our community who are still waiting on research to bring them a treatment. Working with Champions at every level, NNPDF remains dedicated to advocating for patients and caregivers and we will not stop until all people affected by all types of Niemann-Pick disease have access to the care they need.

Throughout October, we invite all of the members of this amazing community to join with NNPDF in recognizing and celebrating the #NiemannPickChampions in your life. Learn how to use our custom-designed template to tell the world about your Niemann-Pick Champion. Create your own social media profile frame to show your support. Visit our awareness month website page to learn more about how you can be an advocate and get involved.  We call our community leaders and dedicated individuals “Champions” because together we can win this battle and achieve our mission of bringing a future of better health and hope to every member of the Niemann-Pick disease community.

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Message from the Executive Director

Jill Beirl

Who’s your #NiemannPickChampion?

October is Niemann-Pick Awareness Month! These next few weeks are an especially important time to tell your family stories on your experiences with Niemann-Pick Disease. Show everyone who’s your #NiemannPickChampion! There is no better time to increase awareness of Niemann-Pick Disease and to educate friends, policy makers, clinicians, and others about this rare disease.

The Niemann-Pick community is filled with champions – champions impacted by this disease everyday, fighting for approved treatments, working towards a cure, and champions keeping the memories of those who have passed.

Over the course of the month, we will offer a range of activities to highlight the community’s #NiemannPickChampion(s) and to promote Niemann-Pick awareness through educational information and social media posts you can share, virtual forums to connect with NNDPF staff and Board members for community conversations, awareness events with our pharmaceutical partners, and of course, we will join with our INPDA partners on October 19th for Global Niemann-Pick Awareness day.

Receiving a rare disease diagnosis can be a devastating and emotional experience. NNPDF is dedicated to an enduring patient support community that embodies strength, hope and empathy for those affected by all Niemann-Pick disease types. We work tirelessly to support and advocate for access to treatments and supports that can make a positive difference in helping affected individuals and caregivers manage a wide range of emotional, financial, and medical challenges. Through our work, families know they are not alone and that an entire community of caring people is  standing alongside them in their journey.

For over 30 years, NNPDF has been a leader in advocating for the needs of all members of the Niemann-Pick community, in support of every #NiemannPickChampion, by connecting our voice with leaders in research, industry, and government at every level who can  help make a significant difference in the lives of those impacted by Niemann-Pick disease.  The battle continues, but we will never give up the fight.

Warm Regards,


Joslyn Crowe, MSW, MA
NNPDF Executive Director

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September 2022 Newsletter

Jill Beirl

Message from the Executive Director  |  Niemann-Pick Awareness Month  |  Community Connections Opportunities  |  Family Support & Medical Conference Recordings  |  Cyclo Therapeutics Patient Newsletter  |  Orphanet Publication Alert  |  ASMD Accelerate  |  LinkedIn Pulse: US Approval of Xenpozyme  |  CheckRare: FDA Approves Olipudase Alfa  |  Fundraising  |  Social Services Resource  |  Surveys & Studies  | Membership   |  Emergency Hardship Program

Message from the Executive Director

Who’s your #NiemannPickChampion?

October is Niemann-Pick Awareness Month! These next few weeks are an especially important time to tell your family stories on your experiences with Niemann-Pick Disease. Show everyone who’s your #NiemannPickChampion! There is no better time to increase awareness of Niemann-Pick Disease and to educate friends, policy makers, clinicians, and others about this rare disease.

The Niemann-Pick community is filled with champions – champions impacted by this disease everyday, fighting for approved treatments, working towards a cure, and champions keeping the memories of those who have passed.

Over the course of the month, we will offer a range of activities to highlight the community’s #NiemannPickChampion(s) and to promote Niemann-Pick awareness through educational information and social media posts you can share, virtual forums to connect with NNDPF staff and Board members for community conversations, awareness events with our pharmaceutical partners, and of course, we will join with our INPDA partners on October 19th for Global Niemann-Pick Awareness day.

Receiving a rare disease diagnosis can be a devastating and emotional experience. NNPDF is dedicated to an enduring patient support community that embodies strength, hope and empathy for those affected by all Niemann-Pick disease types. We work tirelessly to support and advocate for access to treatments and supports that can make a positive difference in helping affected individuals and caregivers manage a wide range of emotional, financial, and medical challenges. Through our work, families know they are not alone and that an entire community of caring people is  standing alongside them in their journey.

For over 30 years, NNPDF has been a leader in advocating for the needs of all members of the Niemann-Pick community, in support of every #NiemannPickChampion, by connecting our voice with leaders in research, industry, and government at every level who can  help make a significant difference in the lives of those impacted by Niemann-Pick disease.  The battle continues, but we will never give up the fight.

Warm Regards,


Joslyn Crowe, MSW, MA
NNPDF Executive Director

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Niemann-Pick Awareness Month

October is Niemann-Pick Awareness Month! Support One Another and Support Your Community by raising awareness and educating others about Niemann-Pick Disease in your community. With your help in spreading awareness, we will make a difference for families at all stages of the Niemann-Pick journey and help us deliver hope!

This month we want you to share your #NiemannPickChampion! Learn how to use our template to make and share your Niemann-Pick Champion and make your own social profile frame to show your support of your Niemann-Pick community.

Visit Niemann-Pick Awareness Month webpage to learn more about how you can be an advocate for those affected by Niemann-Pick disease by spreading awareness in your own community.

   

Community Connection Opportunities

Connect with NNPDF Families, Board of Directors, and Family Services Manager, Laurie Turner during Niemann-Pick Awareness Month. NNPDF will be hosting several options for gathering and connecting virtually starting on October 5th!

Please join us for Coffee and Catching Up and Community Connections: Catching Up virtual gatherings. We look forward to sharing time together – to take a break and reconnect with other Niemann-Pick community members. Be sure to register in advance.

Family Support & Medical Conference Recordings & Photos

THANK YOU to all that joined us in-person and virtually for our 30th Annual Family Support & Medical Conference! We were so happy to see all your smiling faces. It was a wonderful time of sharing, supporting and learning.

For those that were unable to join the conference or if you’d like a review, the 2022 family conference recordings and photos are now available. 

Our Family Support & Medical Conference was a success due to many supporters: from our sponsors and speakers, to our board, staff and volunteers. We THANK YOU for all that you have done to support our family conference.

Cyclo Therapeutics Patient Newsletter

Featuring the Powerful Story of Dee Reynolds

Cyclo Therapeutics’ recently published their patient newsletter featuring Dee and Dan Reynolds. Dee was diagnosed with NPC when she was in her 40s after many years of unexplained symptoms. She and her husband Dan are very well-known advocates – and friends – in the Niemann-Pick community. Here they answer some questions about receiving a diagnosis of NPC later in life and give some advice to families who may have similar journeys. Thank you for sharing your story.

Orphanet Publication Alert!

Our peer-reviewed journal article titled “Health insurance literacy and health access barriers in Niemann-Pick disease: the patient and caregiver voice” was recently published in Orphanet. NNPDF were co-authors on this publication along with Dr. George Diaz based on the large research study we completed previously. Perfect timing in the same week as Xenpozyme’s FDA approval for the treatment of ASMD!

This publication will be a useful resource for payers and decision makers as we look to ensure availability, access, and reimbursement for Xenpozyme now, as well as in the future for our NPC therapies.

ASMD Accelerate is Still Enrolling!

Wylder Nation and NNPDF are still enrolling for ASMD Accelerate! Every child’s story can make a difference for the future of ASMD care.

This study is an opportunity to contribute to ASMD research from home. Signing up with PicnicHealth takes 5 – 10 minutes – after that PicnicHealth’s team collects your child’s longitudinal medical records on your behalf, securely upload them to your PicnicHealth account, and then de-identifies these records for ASMD researchers.

Visit picnichealth.com/asmd-wylder-nation or email [email protected] with any questions.

LinkedIn Pulse: US Approval of Xenpozyme

NNPDF Celebrates Monumental Milestone with the First U.S. Approval for ASMD:  The National Niemann-Pick Disease Foundation joins with patients and families in the U.S. and the entire global Niemann-Pick community in celebrating this historic advance in research: the first-ever U.S. approval of a treatment for ASMD! For more than 20 years, NNPDF has worked to support patients and families impacted by ASMD (acid sphingomyelinase deficiency) by raising awareness and advocating for research that can lead to life-changing therapies. Read complete article.

 

CheckRare: FDA Approves Olipudase Alfa

FDA Approves Olipudase Alfa To Treat Acid Sphingomyelinase Deficiency in Both Children and Adults:  The U.S. Food and Drug Administration (FDA) has approved olipudase alfa for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD).

ASMD is an autosomal recessive genetic disorder caused by mutations in the SMPD1 gene. That gene encodes for acid sphingomyelinase (ASM), an enzyme that metabolizes sphingomyelin. ASMD is also knows as Niemann-Pick disease types A, A/B, and B. Olipudasae alfa is an enzyme replacement therapy designed to replace the deficient or defective ASM. Read complete article.

Fundraising

Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease.

If you have recently hosted a fundraising event, send us your photos and we’ll share them and details from your event in upcoming newsletters!

Bowie Baysox Benefit

THANK YOU to the friends and family of Mark and Darrile Papier who recently hosted their 16th Annual Bowie Baysox Benefit in memory of their son Dillon. The Baysox Benefit was a success with lots of raffle baskets, jewelry, gift certificates, food, and very exciting game finale with the Baysox losing until the last batter blasted a walk off homerun scoring 2 to win the game! A special guest in attendance – Liam Ferguson – was surprised with throwing the first pitch before Mark Papier.

     

THANK YOU to Stacy Stemmerman, Lane Hemsley, Stacy Tillotta, Vera Stricklin, Natalie Johnson, Kevin Nettesheim and Larissa Ross who recently hosted Facebook Fundraisers for the NNPDF! We truly appreciate your support!

Want to host your own Facebook Fundraiser? It’s easy! Visit facebook.com/fund/NNPDF to get started!

Social Services Resource

The journey with those affected by Niemann-Pick disease can be long and complex with caregivers wearing multiple hats. Little Hercules has provided a tool to search for national, state, regional, private, and public programs that can help you with whatever you need. Search for free or reduced cost services like medical care, co-pay assistance, food, utility assistance, transportation, disability programs, and more. Start your search by entering your zip code. Visit littlehercules.auntbertha.com for more information.

Contact Laurie Turner at [email protected] for any questions.

Surveys, Studies, & Market Research

Be sure to check out our Surveys & Market Research webpage for current survey and study opportunities in the Niemann-Pick disease space. Participating in surveys and studies is important to the advancement of health options for our Niemann-Pick community members. Current surveys and studies are listed below. Contact Laurie Turner at [email protected] for any questions.

NNPDF Membership

Enrolling, confirming, or updating your membership will ensure we have accurate information for you and your family. This will help us to continue to provide you with important notifications and updates from the NNPDF.

Click here to update or enroll today!

For assistance contact Laurie at [email protected] or call 920-542-4038

Emergency Hardship Program

The NNPDF Emergency Hardship Program continues to offer assistance to qualified NNPDF U.S. member families facing a crisis. Funding includes but is not limited to, specialized medical equipment, durable medical goods, utility bills (heating and cooling, electricity, phone, water and sewer), home and car repairs, and bereavement expenses. Click here for complete details and eligibility information.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or [email protected] if you have any questions about this program.

            

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NNPDF Celebrates Monumental Milestone with the First U.S. Approval for ASMD

Jill Beirl

The National Niemann-Pick Disease Foundation joins with patients and families in the U.S. and the entire global Niemann-Pick community in celebrating this historic advance in research: the first-ever U.S. approval of a treatment for ASMD!

For more than 20 years, NNPDF has worked to support patients and families impacted by ASMD (acid sphingomyelinase deficiency) by raising awareness and advocating for research that can lead to life-changing therapies. While we have faced many challenges in the effort to bring new treatment options to patients who desperately need them, today we can see a sign of hope for the ASMD community and for all patients living with Niemann-Pick disease.  

The U.S. Food and Drug Administration (FDA) has announced the approval of Xenpozyme (olipudase alfa) for intravenous infusion in pediatric and adult patients with ASMD. Xenpozyme is an enzyme replacement therapy that has been shown to help reduce sphingomyelin accumulation in the liver, spleen, and lung. The efficacy of Xenpozyme for the treatment of ASMD was demonstrated in a controlled study of 31 patients. Based on results from the study, the FDA was able to conclude that Xenpozyme is effective and that treatment helped to improved lung function and reduced liver and spleen size in patients.

NNPDF would like to take this opportunity to recognize the hard work of many people including regulatory and industry leaders who have made this milestone possible. We applaud Sanofi for their commitment to moving this program forward. We also want to emphasize that this approval would not have been possible without the patients and families who participate in clinical research and who use their voices and stories to raise awareness of the need for treatments and support.

More than anything, this life-changing moment is an example of what we can accomplish when we work together. Hopefully, this will bring new levels of momentum and dedication to promising research moving forward. The Niemann-Pick community is resilient and thanks to the bravery and persistence of patients and families, people living with ASMD in the U.S., Europe and Japan can now have access to an approved treatment.

We are so happy to see this amazing progress and will use this to fuel us on our journey to finding approved treatment options for all people impacted by all types of Niemann-Pick.

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