Newsletters
APPROVAL! | Message from Board Chair | 2022 Family Support & Medical Conference
Family Advisory Working Groups | Family Conference Scholarships
NNPDF Cora Sterling Endurance Award | Joele Ruppert & Joseph Colton ASMD Scholarship
NNPDF Research Fellowship Applications | Family Journey with Ashley Lewis
INPDA Letter to CHMP Members | Rare Difference Maker
In the Spotlight | Fundraising | BENEFIT Act of 2021 | Surveys & Studies
Clinical Trial Updates | Community News Updates | Membership
Emergency Hardship Program | Upcoming Events
APPROVAL!!
We are pleased to share the important news from Sanofi that the ASMD community now has its first and only approved treatment option available for ASMD! Sanofi’s Xenpozyme (olipudase alfa) is now approved in Japan for the treatment of children and adults with non-CNS manifestations of ASMD.
John Reed, MD, PhD, Executive Vice President, Global Head of Research and Development of Sanofi expressed that “today’s approval of Xenpozyme is a watershed moment for ASMD patients and their families, representing 20 years of research and the shared efforts of advocacy partners, clinicians, and patients. As the world’s first medicine approved for ASMD, Xenpozyme offers a potentially transformative option for this historically neglected community.”
NNPDF joins the entire community in celebrating this historic win! Read complete press release.
Message from the Board Chair
This has been a historical week for the ASMD community. On March 28, Sanofi announced that olipudase alfa was approved in Japan for the treatment of pediatric and adult patients with ASMD! This is the first and only approved therapy for ASMD patients in the world! It took a village to help us reach this amazing milestone!
Key contributors include Dr. Edward Schuchman who identified the gene encoding, the enzyme involved in ASMD. He also developed the mouse model for ASMD and the first genetic screening test for ASMD. In addition, he pioneered enzyme replacement therapy for ASMD and collaborated with Sanofi to help bring olipudase to clinical trials. Thank you, Dr. Schuchman!
Our deepest appreciation for all the clinicians, genetic counselors, nurses and professionals that carried out the olipudase alfa trials. The clinical leaders at trial sites around the world included Simon Jones, Maurizio Scarpa, Karl Mengal, Robert Giugliani, Nathalie Guffon, Antonio Barbato and Isabele Batsu. Here in the U.S., we are especially grateful for Drs. Renata Gallagher, George Diaz, and Melissa Wasserstein, along with their amazing clinical teams, for their tireless efforts in not only leading these clinical trials but for providing great care for all our patients. We are so appreciative for your time, your effort, your passion and your leadership.
We would also like to thank all the patients and families who participated in the olipudase clinical trials. In the beginning, it was the original 5 adults who have now all continued to be part of the clinical trial for almost a decade! They have donated hours, days, weeks and years to the community. Through their efforts, we understand the persistent benefit of olidpudase can be seen years after starting therapy. Our appreciation to the pediatric patients and families who have also been involved in the clinical trial for up to 7 years. Lastly, a heartfelt thanks to all the adults who enrolled in the ASCEND trial, a year long trial that required travel to an approved site every two weeks for a year with a 50/50 chance of receiving placebo during the study period. In an ultra rare disease with a limited number of patients, each trial participant’s data, including the placebo data, is critical in demonstrating the benefit of this therapy. Thanks to all!!!
We appreciate Sanofi’s work in the rare disease space and commitment to providing the first approved therapy for all Nieman-Pick patients. We understand that an approval in Japan does not translate to approvals elsewhere. However, it is a tremendous first step in our goal of allowing all patients with ASMD to have the opportunity to receive an approved therapy. NNPDF will continue to advocate for approval in the U.S. and around the world. Thank you all for supporting the advocacy work we do and supporting the NNPDF. Let’s work together to push this approval across the finish line in the coming months.
Read the complete press release.
With Warm Regards,
Justin Hopkin, MD
NNPDF Board Chair
NNPDF Family Support & Medical Conference
in Conjunction with INPDA Biennial Meeting
JOINT CONFERENCE DATES: July 28 – August 1, 2022
NNPDF: July 28 – 30 | INPDA: July 29 – August 1
Wyndham Grand Orlando Resort Bonnet Creek | Orlando, Florida
____________________________________________
Conference Registration | Hotel Reservations | Conference Information
nnpdfconf.org
NNPDF Family Advisory Working Groups
The National Niemann-Pick Disease Foundation, Inc. (NNPDF) will host Family Advisory Working Groups that will take place on Thursday, July 28, 2022 from 1:00 pm – 4:00 pm EST at the Wyndham Grand Hotel, Orlando. This is an optional event that will take place prior to the start of our Family Support & Medical Conference. Pre-registration is required.
Family Advisory Working Groups will provide Industry partners the opportunity to connect privately with a group of 6-10 Niemann-Pick Type C or ASMD patients and/or immediate family members for a 3-hour session. Due to limited space, there is a limit of 2 participants per family. Participants must be adults living in the United States. Participants are eligible for a $350 stipend per participant and 1-night hotel included per family (night of July 27th). Application deadline is May 16th.
Family Conference Scholarships
Scholarship assistance is available to NNPDF registered US families attending the NNPDF Family Support and Medical Conference in Orlando, Florida from July 28 – 30, 2022.
Assistance is available to help NNPDF families living in the US, who are experiencing financial need to help offset the cost of attending the 2022 Family Support and Medical Conference. Family Conference Scholarships are limited to immediate family members of an individual with NPD. The NNPDF Family Conference Scholarship will provide up to three (3) nights hotel (room and tax only*) to eligible applicants. Application deadline is May 16th.
NNPDF Cora Sterling Endurance Award
Do you know a great young person who shines for Niemann-Pick awareness? NNPDF is seeking nominations for the NNPDF Cora Sterling Endurance Award. This award is presented annually at the NNPDF Family Support & Medical Conference to a young person (under age 25) who shines in raising awareness of Niemann‐Pick disease in the community. Nomination deadline is May 31st.
Joele Ruppert & Joseph Colton ASMD Scholarship
Joele Ruppert and Joseph Colton are a much-loved daughter and son, both diagnosed with ASMD. This scholarship has been created in honor of the lives of these two cherished children and their parents who embodied the highest values of the human spirit. Read complete story and learn more about this scholarship opportunity. Application deadline is May 15th.
NNPDF Research Fellowship Applications
NNPDF is accepting Research Fellowship Applications. The Peter G. Penchev Niemann-Pick Type C Research Fellowship and the Edward H. Schuchman ASMD Research Fellowship are 1-year awards intended for early-stage researchers. These awards support young scientists in pursuit of independent hypotheses and encourage scholarly development about Niemann-Pick Disease.
Niemann-Pick Disease research is one of the pillars of our mission and we are proud that since 1993, NNPDF has funded 52 grants and 20 fellowships totaling over $4,000,000. Our fellowships and grants have contributed to the identification of disease-causing genes, insights into the pathogenesis of NPC1 & NPC2, support of the NPC cat model at a critical juncture, and development of an assay for newborn screening. Many former grantees and fellows have remained active contributors in the Niemann-Pick Disease space. Application deadline is June 1st.
Contact [email protected] with questions.
Family Journey with Ashley Lewis
During our 2021 NNPDF Family Support & Medical Conference we were honored to have Niemann-Pick community members share their Niemann-Pick stories with us.
Today we feature Ashley Lewis, Mom of Linwood, sharing her family story of living with ASMD. Thank you, Ashley for sharing your personal story with us. Watch Ashley’s family journey.
INPDA Letter to CHMP Members
The International Niemann-Pick Disease Alliance (INPDA) sent the following letter in support of Orphazyme’s candidate in NPC to the Committee for Medicinal Products for Human Use (CHMP) members at the European Medicines Agency (EMA) in advance of the recent review of Arimoclomol.
THANK YOU to the nearly 500 family members from around the world who signed their support as well! Read the letter.
Rare Difference Maker
NNPDF’s Joslyn Crowe and Laurie Turner were recently featured in Engage Health’s Rare Disease Difference Maker project. This project was created to recognize researchers, physicians, organization leaders, patients, parents, teachers, friends, and many others who have contributed immensely to the rare disease space. Read their story.
João Paulo De Souza
Sr. Program Manager, AllStripes
Tell us a bit about yourself, such as where do you live and what do you enjoy doing?
My name is João Paulo – I know, it’s a bit of a tongue twister, but most people call me JP. I’m a Bay Area native who returned after living in San Luis Obispo, California. From an early age, I knew I wanted to help people. After graduating from the University of California, Berkeley, most of my work has been in a variety of clinical and biotech settings: from creating treatment plans for children with varying developmental disorders to using AI for blood diagnostics.
These days, I’m a Sr. Program Manager at AllStripes, where our mission is to unlock new treatments for people affected by rare disease. I’m fortunate to work alongside many dedicated and talented individuals working toward the same mission, and seeing amazing work being done for communities like NPC. During my free time, I take advantage of the many hikes and parks the Bay Area has to offer with my best friend Samson, my 4-year-old Goldendoodle. I love to experience different cultures through travel, and of course, using my taste buds.
What caused you to get involved in the Niemann-Pick community?
The NPC Sibling Study was one of the first projects I started helping with when I joined the team here at AllStripes. Since then, I’ve had the privilege of working closely with NNPDF, Firefly Fund, Niemann-Pick Canada, and the Ara Parseghian Medical Research Fund. Our work has allowed me to connect with members of the community and hear such unique and powerful stories, which continue to drive me each and every day.
How does the NPC Sibling Study support early NPC intervention?
The intent of the Sibling Study is to provide evidence to support the inclusion of NPC in newborn screening (NBS) programs. Achieving this will allow physicians to diagnose babies with NPC early, which will allow for early intervention. Early intervention may increase the effectiveness of treatments and improve quality of life. Getting NPC added to newborn screening panels would be a huge accomplishment and success for the community!
Why are sibling pairs needed for this research?
In short, the Sibling Study is attempting to show the effects of early intervention. How will this research project do this? When two siblings have the same diagnosis and one sibling had intervention earlier than the other, researchers can determine whether intervening early leads to slower symptom onset and/or decreased severity of symptoms.
Why is data from families whose loved ones have passed important to the study?
While there’s currently no cure for NPC, there have been advancements in treating it. Some of these advancements are recent enough that, in some cases, people who have died from NPC did not have the chance to be treated with disease-specific therapies. Comparing medical records from deceased patients to medical records from living patients can help provide evidence that early diagnosis and intervention makes a real difference. This means the records of loved ones who have passed away from NPC are especially helpful for researchers trying to build evidence for adding NPC to newborn screening.
What are your hopes for the future of the Niemann-Pick community?
My hope is that our work with the NPC community can support the addition of NPC to newborn screening panels. Beyond that, our goal is to unlock new treatments for the NPC community. There are many like me who are working toward this goal, and the Sibling Study is just the first study we hope to work on for the NPC community.
How can families get involved?
To join other patients and families on AllStripes go to allstripes.com/npc.
If anyone has any questions about the study of AllStripes, please feel free to email [email protected].
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 the Brighton Basketball community who recently held a Super Bowl Square Fundraiser to help raise awareness of Niemann-Pick disease and funds for the NNPDF.
“This foundation is important for our community as we look forward to supporting Garrett Hopkin and his family. Garrett lives with Nieman-Pick Disease and is our biggest fan! He is a current student at FRES and has siblings in both the boys (Grady) and girls (Avery) basketball programs.”
THANK YOU to Dawn Cooley, Carol Anderson, Jennifer Gregory, Mari Lombardo Sanetra, and Kayla Miller 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!
BENEFIT Act of 2021
NNPDF recently signed on to a letter to US Senators and Representatives in support of the BENEFIT Act of 2021. The Better Empowerment Now to Enhance Framework and Improve Treatments Act of 2021 (BENEFIT Act of 2021) is a bill that requires the US Food and Drug Administration (FDA) to consider relevant patient-focused drug development data, such as data from patient preference studies and patient-reported outcome data, in the risk-benefit assessment framework used in the process for approving new drugs. Read the letter.
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.
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 Sanofi:
We are pleased to share an important update from Sanofi: Xenpozyme® (olipudase alfa) APPROVED in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency (ASMD). Read the press release.
Update from Orphazyme:
NNPDF has received the following communication from Orphazyme regarding this week’s press release on arimoclomol programs for NPC. Read the letter and press release.
Community News Updates
Announcement from Azafaros:
Azafaros announces FDA Grant of Orphan Drug Designation for AZ-3102 in the treatment of Niemann-Pick Disease type C. Read complete announcement.
INPDA Letter on Arimoclomol to CHMP Members
The International Niemann-Pick Disease Alliance (INPDA) sent the following letter to the CHMP members at the European Medicines Agency (EMA) in advance of the upcoming review of Arimoclomol. Thank you to the nearly 500 family members from around the world who signed their support as well! Read the letter.
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.
Dear NNPDF Community,
Participation is easy!
NNPDF is now accepting Research Fellowship Applications.
When did you receive Caleb’s diagnosis and what to led to this?
Dear NNPDF Community,
As I look forward to the New Year, I am so encouraged by bravery and resilience of the many families that work with us. I believe our community is stronger than ever. Throughout 2021, we had some signs of progress, and we faced some setbacks when promising clinical development programs and trials were either cancelled or failed to win FDA approval. In response, we saw our community join together to fight even harder to address the issues that are important to us. I am inspired by so many examples of bravery and determination. Dale Carnegie once said, “Most of the important things in the world have been accomplished by people who have kept trying when there seemed to be no hope at all.” Our community is continuing to fight for our families despite our challenges. We are closer than ever to having approved treatments in Niemann-Pick disease. And together we will make a positive difference.
Mom of Linwood, ASMD
What led to and when did you receive the Niemann-Pick disease diagnosis?
Patient AirLift Services is a nonprofit that arranges free flights for medical patients requiring medical diagnosis, treatment or follow-up who cannot afford or are unable to fly commercially.
THANK YOU to our Niemann-Pick community for participating in Niemann-Pick Awareness Month! You shared your stories, promoted our posts, held fundraisers, and continued to spread awareness of Niemann-Pick disease throughout the month of October. Thank you to our donors for supporting our organization. TOGETHER we can make a difference!
Boston Children’s Hospital
How did you come to be involved with NNPDF?
Orphazyme Update on Arimoclomol
Wisconsin Rare Disease Advisory Council
Thank You
On August 3rd, another NPC Community Listening Session was held with the FDA. Thank you to our clinician and family speakers who participated. The Summary Report is now available and can be found on our website. We continue our outreach on these urgent topics with the FDA to ensure the patient and Niemann Pick community voice is heard. 
An NPC virtual Capitol Hill Briefing is being organized by the NPC moms in coordination with Rep. Lesko of Arizona and Rep. Sewell of Alabama. The event will take place on 
Health care workers play an important role in the lives of our Niemann-Pick families and are valued throughout our community. NNPDF is offering to send out Thank You cards to our Niemann-Pick health care workers to let them know they are very important to us. To participate, 
In this presentation, Amy Aikins, Director of Government and Social Programs at the Little Hercules Foundation provides an overview of waiver programs, why you might be interested in enrolling your child, how to find out what waiver programs may be available, and tips for those who are already enrolled in a waiver program.
NNPDF Board Members are essential in the progress of Niemann-Pick Disease awareness and your NNPDF organization. They volunteer selflessly to serve in these vitally important roles, giving their time and energy to keep your NNPDF family support organization moving forward. We truly appreciate and THANK each and every one of you for all you do.
Tell us a bit about yourself, such as how old you are and what do you enjoy doing.
How do you talk to your friends about Niemann-Pick?
I would say to them, don’t worry, people will help. There are people who have been studying in this field for years, and they know what it’s like. You can ask anybody for help, people who have little siblings with NPC, and you can ask your parents. You can ask your parents why it just happened to them and not you. I always treat Marian like a sister who doesn’t have Niemann-Pick Type C – I never treat her like she always needs help, we do all the normal things sisters do, fight with each other and then make up having cookies. Whenever we drink milk, we would have silly mustaches and then say, “you have a milk mustache!” And then May May would say awful jokes, like, “how do cows say, moo” and then she says, “moo.”
In this highly insightful presentation, Bill Berry, Principal at Berry & Company, shares his experience and expertise in how to communicate your story with various media outlets, letting them know that they SHOULD cover your stories on the very important issue of Niemann-Pick disease.
Dr. Justin Hopkin, NNPDF’s Board Chair was honored with Sanofi Genzyme’s annual TORCH Award on Thursday, August 26th. Justin has been a champion in representing and advancing the needs of the entire Niemann-Pick community. He has gone above and beyond while facing his own personal experiences and challenges with his son Garrett (ASMD). It is his compassion, dedication, determination, and commitment to the entire Niemann-Pick community that we are honored to have him advocating for our cause.
To be eligible for funding, NPD individuals and families must reside in the US and must be enrolled as a member of the NNPDF for a minimum of 6 months prior to requests*. Funding is available for up to $1000 per calendar year per eligible member. Families with multiple children with NPD are eligible to apply for each affected individual. The NNPDF Board of Directors and Emergency Hardship Program Committee members work to keep the fund fiscally sound, by being the last resort to be used, limiting regular, recurring use by any one recipient, and encouraging donations.
NNPDF is forming a new Advocacy Committee for community members who want to directly help with input and planning of advocacy initiatives. Your voice is important to getting our message out that our Niemann-Pick community needs approved medicines now. Contact 
Joele Ruppert and Joseph Colton are a much-loved daughter and son, both diagnosed with ASMD, whose parents, Jeff and Kara Ruppert and James and Alexandra Colton, demonstrated compassion, courage, selflessness, and perseverance from the beginning to the end of their children’s journeys. The words and deeds of these two families proved inspirational to another family of a child diagnosed with ASMD, and indeed, to all who knew Joele and Joseph. This scholarship is in honor of the lives of these two cherished children and their parents who embodied the highest values of the human spirit.
The Joele Ruppert and Joseph Colton ASMD Scholarship fund began in August 2020 when Evren Ayik, at that time a graduating high school senior, was awarded the Sanofi Genzyme TORCH Award for his work in advocacy for individuals and families affected by ASMD. The NNPDF, in gracious acknowledgement of Evren’s wishes to endow a scholarship for other students with ASMD, supported his proposal for the financial contribution that is offered to the TORCH Award recipient’s chosen non-profit organization serving the rare disease community. In naming the scholarship, Evren recognized that many years earlier, he and his own family were also encouraged and inspired by the Ruppert and Colton families, and he wished to honor both of the children who lived with ASMD, Joele and Joseph, and their respective parents, all of whom are exceptional people.
JP Honsinger was diagnosed in 5th grade with NPC. On June 25th he graduated from high school with his class of 800 students! JP will attend the Adult Transition program in the fall for the next three years. Congratulations JP!
Kaila Guy, ASMD, was the Xcel Platinum Florida state gymnastics champion on Floor and came in 2nd in the all around. Kaila qualified for regionals in Atlanta, and placed 3rd on floor and in the all around. Congratulations Kaila!
Following a review of our Clinician Reported Database involved clinicians, researchers, industry representatives, the INPDR are pleased to report that we have completed the NPC database amendment with ASMD to follow soon after. The new database will be rolled out over the coming weeks to our 18 existing sites and will now include data on adverse events and co-morbidity. There are a further 47 sites that are currently in the pipeline for onboarding to the INPDR with the potential to capture data for more than 1000 patients internationally. While onboarding and recruitment has been impacted by the COVID pandemic, the registry has made important changes to accelerate recruitment. This includes facilitating postal consent so patients and families do not need to attend clinic to decide to take part in the CRD. The registry has also translated our documents into ten languages which addresses another significant barrier to registry recruitment.
NNPDF has been asked to share the following ASMD market research interview opportunity. Evidera is seeking to conduct phone interviews with adults with ASMD to better understand the ASMD journey.
The ASMD Accelerate study continues to enroll both patients with Infantile Neurovisceral ASMD (NPA) and Chronic Neurovisceral ASMD (NPA/B).
NNPDF, APRMF, NPCanada and Firefly Fund Seek Your Participation in a first-of-its-kind Newborn Screening Research Study
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 
Adrabetadex Update:
NNPDF has created a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. 
The NNPDF Emergency Hardship Program offers help 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. This assistance program provides relief to members facing a situation that threatens their immediate health and safety, or that causes a terrible hardship. It is intended to provide short term financial assistance in a crisis situation and is not intended to address long term financial challenges.
Patient AirLift Services is a nonprofit that arranges free flights for medical patients requiring medical diagnosis, treatment or follow-up who cannot afford or are unable to fly commercially.
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. 
Dear NNPDF Community,
Deadline for nominations is May 31st. Do you know a great young person who shines for Niemann-Pick awareness? NNPDF is seeking nominations for the NNPDF Cora Sterling Endurance Award. This award is presented annually at the NNPDF Family Support & Medical Conference to a young person (under age 25) who shines in raising awareness of Niemann‐Pick disease in the community. 
This month, May 2021, for the very first time ever, a newborn was screened for Niemann-Pick Type C. 
My name is Lori McKenna Gorski and I am delighted to have joined Cyclo Therapeutics to oversee Patient Advocacy and Engagement. Most of my career has been focused on working in biotechnology, and particularly in rare diseases. I spent many years at Genzyme (now Sanofi Genzyme) in roles in both communications and patient advocacy, including on the Niemann-Pick B/ASMD program. I got to know your global community before the clinical trial commenced, and through such disease awareness programs like Expression of Hope.
We are continuing to request that ALL current and interested community members 
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 
Mallinckrodt Pharmaceuticals has reached an agreement to divest and transfer the Investigational New Drug (IND) application for experimental drug adrabetadex (VTS-270) to Mandos, LLC. 
The ASMD Accelerate study continues to enroll both patients with Infantile Neurovisceral ASMD (NPA) and Chronic Neurovisceral ASMD (NPA/B).
Patient AirLift Services is a nonprofit that arranges free flights for medical patients requiring medical diagnosis, treatment or follow-up who cannot afford or are unable to fly commercially.
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. 
Dear NNPDF Community,
NNPDF hosted an FDA listening session on Niemann- Pick Disease on Friday, April 9th. The listening session was a platform for the NNPDF to present to the FDA/CDER and to share the concerns and priorities from the entire Niemann-Pick disease community. To review the NNPDF FDA Listening Session Summary Report, please 
Thank you to all that have recently updated their membership. We are continuing to request that ALL current and interested community members confirm or update your membership information to ensure we have accurate contact information for you and your family for continued important notifications from the NNPDF.
The Newborn Screening Working Group will hold its first virtual meeting in 2021 on Friday May 14th at 4pm ET. If you are interested in learning more about the efforts of the Newborn Screening working group and the progress that is being made towards realizing the goal of having NPC added to the RUSP (Recommended Uniform Screening Panel), or if you would like to join this important advocacy and research project, please contact Pam Andrews at Firefly Fund (
NNPDF has created a listing of medical institutions that have experience in caring for and treating Niemann-Pick disorders. This project is supported by our Scientific Advisory Board and we are proud to offer this valuable resource to our families as they search for medical care and treatment. This information has been provided to us by the institutions. 
HEY SUPERHEROES!
NNPDF Community,
Wylder Nation Foundation has recently partnered with PicnicHealth to launch the ASMD Accelerate study as a way for families to easily contribute to advancing research for ASMD. 
Plans are well under way for the NNPDF-INPDA Family Support & Medical Virtual Conference taking place July 29 – August 1st! Mark your calendars and watch for registration details coming soon! In the meantime, remember to download the 
When did you receive the girls’ diagnosis? What led to diagnosis?
What caused you to get involved with NNPDF initially and how has being an NNPDF member benefited your family?
Thank you to everyone who contacted us with feedback for the INPDR patient, families and friends’ newsletter. There’s still time to share your opinions so please get in touch through email at 
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