Newsletters
Message from the Executive Director
Dear NNPDF Friends and Family,
Rare Disease Day is approaching. On February 28th, families from around the world join together as one voice. Throughout the month, Niemann-Pick families across the US sent photos in support of our “I Care About Rare” program and helped us amplify the Niemann-Pick voice.
Niemann-Pick is one of nearly 7,000 rare diseases. The path to diagnosis for a rare disease can be long, winding, and complicated with few specialists and supports. At the same time, the path to approval of new medications for rare diseases is complicated. We have less patients for clinical trials than a “typical” disease and traditional clinical trial designs may not fit the unique patient populations of rare diseases like Niemann-Pick.
NNPDF is advocating on behalf NPD families, to treat rare diseases differently from mainstream diseases with larger population. We are sharing our thoughts and concerns with the FDA in order to discuss challenges of clinical trials with small patient populations, to find solutions that may make approved treatments possible in the near future, and to increase the patient voice in decision making.
To help support our positions, we now have a vast array of data from our “Challenges of Access” Survey conducted in 2020. Preliminary findings were presented in a poster at WORLD Symposium and will also be shared in a summary report shortly. Thank you again to all of the families who participated in this survey. Your responses are helping us to better understand the health insurance needs and challenges families face in accessing care and services. The data we gathered will provide the foundation needed to discuss these challenges with key opinion leaders, like legislators and regulatory officials, and to provide a platform for our fight to ensure coverage for treatment options.
Last summer our Family Support & Medical Conference featured a keynote address by Dr. Kathleen, Acting Director, Division of Rare Diseases and Medical Genetics (DRDMG), Office of New Drugs, US Food & Drug Administration. This summer, our 2021 Family Conference will again take place as a virtual event. Our Scientific Advisory Board recommend a best way to safely gather our Niemann-Pick families for our annual conference is to hold a virtual conference this year. Our Board of Directors and staff take this recommendation, and the health of the Niemann-Pick community, seriously and support this guidance.
Our program will still be held July 29th – August 1st – starting with Family Advisory Working Groups on the morning of July 29th, and our conference beginning on the evening of July 29th. We are fortunate to host the INPDA Global conference in conjunction with ours and are excited to be joined by our international friends and families. Given the success of last year’s virtual conference, we know this year will be another interactive and engaging weekend filled with clinical & research updates and discussions, our unique family break-out sessions, and plenty of time to connect on our virtual conference platform throughout the weekend.
Registration will be available shortly. Watch for the announcement and link soon! In the meantime, don’t forget to download the NNPDF app for connecting, updates, info, contests, and more!
Warm Wishes,
Joslyn Crowe, MSW, MA
NNPDF Executive Director
Rare Disease Day
Rare Disease Day is February 28th! Show your support on Rare Disease Day by raising awareness of what it means to be rare. Building awareness of rare diseases is so important because 1 in 20 people will live with a rare disease at some point in their life. Despite this, there is no cure for the majority of rare diseases and many go undiagnosed. Rare Disease Day improves knowledge amongst the general public of rare diseases while encouraging researchers and decision makers to address the needs of those living with rare diseases.
Anyone can get involved in Rare Disease Day awareness. Participants can find helpful resources, activities and events to get involved at rarediseaseday.org. This global event was founded by EURORDIS and is sponsored by the National Organization for Rare Diseases (NORD).
NNPDF Rare Disease Day Participation
Rare Disease Day Facebook Profile
To raise awareness for Niemann-Pick disease and Rare Disease Day the NNPDF has created a custom frame for your Facebook profile pictures. Also, please feel free to display and share the NNPDF Rare Disease Day Facebook banner to promote awareness.
To add the NNPDF Rare Disease Day frame to your Facebook profile picture:
- Go to your Facebook page and click on your profile picture.
- Click on “Add Frame”.
- Search for “NNPDF Rare Disease Day 2021” frame and click your selection.
I Care About Rare
Show us why YOU Care about Rare! During the month of February, join us in promoting awareness of Rare Disease and Niemann-Pick disease.
- Click here to print out the I Care about Rare sheet. Add WHY you Care About Rare, snap a photo, and share your on your social networks with the hashtag #NiemannPickRare – let’s flood our social pages with photos of why you Care about Rare and Niemann-Pick disease!
- Send your photos to [email protected] with RDD in the subject line to be included in our Rare Disease Day slideshow with your I Care About Rare photos!
Membership Update
Dear Friends,
As NNPDF Board Chair, I am honored to write you on behalf of the National Niemann-Pick disease Foundation. I hope this finds you well during these most unusual times.
We are on the cusp of a tremendously exciting period and hope to see the first approved therapies to treat Niemann-Pick disease. In an effort to best serve you and keep you updated, NNPDF has recently upgraded our data management system to meet the growing needs of our organization. We ask ALL current and interested community members to confirm or update your membership information in this new system by March 15, 2021 to ensure we have accurate contact information for you and your family.
Membership is complimentary for Niemann-Pick families, friends, and community supporters. Your NNPDF membership enrollment will ensure continued notifications of upcoming opportunities to connect with families, participate in advocacy efforts, learn about research breakthroughs and fellowship funding, participate in a clinical trial, and honor a loved one.
Enrollment also includes NNPDF member-only benefits such as:
- Emergency Hardship Program
- Holiday Gift-Giving
- Family Conference Scholarships
- Navigating Together family support sessions
Confirm or update your membership information by March 15, 2021
for your chance to win $50 towards NNPDF Gear!
For enrollment assistance contact Laurie at
[email protected] or call 603-413-8707
As a member, we’ll keep you updated along the way as information becomes available for clinical trials, treatments, and advocacy efforts by way of email and our enhanced social media presence.
NNPDF Staff and Board of Directors are available and eager to talk with you and help you in any way we can. We are here for you and want to hear what is important to you! Please reach out to Laurie Turner, Family Services Manager at [email protected] or 877-287-3672 for any questions or for assistance in updating your information.
Thank you for your support and I look forward to our continued journey together.
Justin Hopkin, MD
NNPF Board Chair
NNPDF Family Support Medical Conference Update
Dear Friends and Families,
NNPDF’s Scientific Advisory Board recommend a best way to safely gather our Niemann-Pick families for our annual conference is to hold a virtual conference this year. Our Board of Directors and staff take this recommendation, and the health of the Niemann-Pick community, seriously. We have decided that our Family Support and Medical Conference taking place this summer will be a virtual event.
Our program will still be held July 29th – August 1st – starting with Family Advisory Working Groups on the morning of July 29th, and our conference beginning on the evening of July 29th. We are fortunate to host the INPDA Global conference in conjunction with ours and are excited to be joined by our international friends and families. Given the success of last year’s virtual conference, we know this year will be another interactive and engaging weekend filled with clinical & research updates and discussions, our unique family break-out sessions, and plenty of time to connect on our virtual conference platform throughout the weekend.
Registration will be available shortly. Watch for the announcement and link soon! In the meantime, don’t forget to download the NNPDF app for connecting, updates, info, contests, and more!
Save the Date for 2022: We still gather in Orlando, along with our INPDA friends and families in late July 2022.
NNPDF In Action
WORLDSymposium 2021
Data from NNPDF’s Challenges of Access survey was presented at this month’s WORLD Symposium as a poster (pictured below). The survey aimed to understand health insurance literacy and challenges accessing health services in Niemann-Pick disease through the eyes of patients and families and was the most comprehensive survey conducted of the Niemann-Pick community to date. Significant findings showed that Niemann-Pick disease families had a higher level of heath literacy than the general population however insurance literacy gaps included insufficient knowledge of health insurance terminology and difficulty calculating expenses based on insurance coverage. The results showed that NNPDF has a role to play in better helping families understand Medicaid waivers, plan deductibles and out-of-pocket maximums.
The survey also showed the most common life impacts from access challenges in the Niemann-Pick community were mental health and physical symptoms. Caregiver’s most frequent burden were caregiver burnout and concerns about advocating in the right way. Results shows that Niemann-Pick community members most feared delays in access or being unable to access medications, services, and providers.
The full results of the survey will be published in a final report which will be shared publicly. The results of this research will be used to shape NNPDF’s advocacy efforts to improve patient outcomes.
Navigating Together: Support for all phases of Niemann-Pick disease
NNPDF Family Services is pleased to share that the first Navigating Together small group session has begun. Navigating Together: Legacy Families started this week to offer support to our members that have lost a loved one. NNPDF recognizes our bereaved families are an integral part of our community and we hope to offer support with our Navigating Together sessions.
Navigating Together series is a new direct services program to provide a confidential space for families to process the complexities related to the realities of loss on multiple levels and is led by a licensed professional trained in grief counseling.
Additional Navigating Together sessions will be held for ASMD and NPC Families and may include specific sessions for families during all phases of the NP journey such as – late Onset NPC, Legacy Families, Newly Diagnosed Families.
If you have any questions or would like more information about Navigating Together, please contact Laurie Turner, Family Services Manager at [email protected] or 603-413-8707.
Thank You to all that generously supported the NNPDF with your financial gifts in 2020 through your donations, family fundraisers, and social media fundraisers. It is through the generosity of donors like you that we are able to provide vital unmet services to patients and families along their Niemann-Pick journey. Your support matters. We can’t do it without you! Your ongoing commitment to our organization is greatly appreciated.
Volunteers Needed
Thank you to those of you that have shown interest in volunteering for the NNPDF! Your time is invaluable to our NPD community. We are currently collecting resumes and letter of interest from those interested in volunteering skills and time to assist with focus groups, committee teams and even as a Board member. If you are interested in becoming a NNPDF volunteer, please let us know at [email protected] and we will follow up with you with the process for applying.
Please send all letters of interest by March 15th.
Coffee & Catching Up
JOIN US for Community Connections Coffee & Catching Up on Monday mornings at 11am EST! Connect with other Niemann-Pick community members who share your journey. Click here to register.
If you have any questions please contact Laurie Turner, Family Services Manager at [email protected] or 603-413-8707.
ASMD Update
Sanofi Genzyme is excited to announce the launch of a new educational phone line for the Acid Sphingomyelinase Deficiency (ASMD) community. ASMD is historically known as Niemann-Pick disease type A, A/B, and B. This phone line is staffed by patient education liaisons (PEL) who are available to speak with callers about ASMD symptoms, diagnosis, and other disease-related topics.
You can speak with an ASMD PEL Monday – Friday, 8 am – 8 pm ET by calling 1-800-745-4447, Option 3 for Patient Services, then press 1 “for more information on ASMD.”
This number and more ASMD-related information can also be found at www.ASMDFacts.com
MAT-US-2100595
INPDR Update
This month, the INPDR launched the first of a quarterly newsletter for professionals working with the Niemann-Pick disease community. The newsletter is an opportunity to keep readers informed of our operational progress, as well as recruitment updates. We will also be covering events and projects in the pipeline. A separate newsletter especially for patients, their families, friends and carers will be launched early in the Spring.
We need your help to make sure the newsletter is what you want it to be and tells you what you want to know. The team behind the INPDR are so excited about the difference our work can make to progress and the future for the Niemann-Pick community across the world but what really matters is what patients and their families think. Patients are at the heart of everything we do so it is our hope the newsletter will inform and inspire. What would you like to know?
There are no silly questions and over the coming months we will try to cover everything you want to know: How easy is it to sign up? Maybe you don’t understand what a patient registry like us is for? Perhaps it’s hard to see what difference our work will make? When your To-Do list is as long as your arm, why should you care about the INPDR?
Please feel free to get in touch through email ([email protected]), visit inpdr.org/contact-us/ or social media channels with questions, thoughts and ideas – whichever way you feel comfortable sharing your thoughts, we’d love to hear them.
You can also sign up to receive our quarterly newsletter by clicking here.
Emergency Hardship Program
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.
If you have questions about this program, please contact Laurie Turner, Family Services Manager at 603-413-8707 or [email protected]. Applications will be reviewed by the NNPDF Emergency Hardship Program Committee consisting of 2 Board members and 3 members-at-large.
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.
Download and complete fillable PDF application, and return via email to Laurie Turner at [email protected].
*Those with new diagnoses are eligible to apply if diagnosis was within 6 months of request.
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 Cyclo Therapeutics: Cyclo Therapeutics Presents Positive Data from Clinical Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021. Click here for complete announcement.
Update from Orphazyme: Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium. Click here for complete announcement.
Illustrate Your Imagination
HEY SUPERHEROES!
In recognition of Global Niemann-Pick disease Awareness Month, the Orphazyme team invites you to join us on a quest to “Illustrate Your Imagination.”
Our team often hears brave stories of your experiences living with Niemann-Pick disease and times when you took on challenges with deep personal courage, perseverance, care, and integrity. We’d like to help your super stories take flight.
Your mission, should you choose to accept it: Visit IllustrateYourImagination.com and download a kit to create your own comic. The kit is filled with page templates, instructions, and some flair to help bring your story to life. Submit your story to our online comic book gallery and check out submissions from other superheroes around the world.
We can’t wait to see the creative stories you come up with!
Regan Sherman
Head of Global Patient
Advocacy Relations
Orphazyme
NORD and partners recently hosted a 
By Pam Crowley Andrews, Executive Director, Firefly Fund and Allison Rosen, 3D Communications for Firefly Fund
Several NNPDF Community members had help in making their holidays a little brighter thanks to an anonymous special donor. Our donor family wished to help ease the burden of Holiday stress, knowing that living with NPD can be difficult and 2020 came with its own set of difficulties. THANK YOU to our donor family – “…you all made us very happy with your act of love.”
When did you learn about Garrett’s diagnosis? What were your initial thoughts and concerns?
What have you been able to do in your role as a grandparent?
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 
by Miranda Feinberg, NNPDF Intern
One way that Serina focuses on the way grief affects holidays or special occasions is how yearly traditions become disrupted or hollowed. What once might have been enjoyed every year and associated with specific holidays no longer feels special. The tradition is no longer the same; something is missing, someone is gone. Traditions and routines are so ingrained in our everyday lives, so when some part of them is changed and disrupted, the tradition itself becomes troublesome and harmful to our everyday lives. Furthermore, the special-ness of holidays and the magic that is tied to them can conflate the importance of traditions in a way that makes the loss of such traditions even more distressing. Serina uses Christmas as an example of a special occasion with many long-held traditions that can be irrevocably affected by loss. She describes how, after loss, the winter snow transformed from exciting and magical to gloomy and cold, how putting up the Christmas tree became a chore, and how holiday parties became something to be avoided rather than attended.
The loss of a loved one is felt deeply at those times where there was once so much happiness. The loss of that person becomes tied to the loss of those traditions. The simultaneous feelings of grief and sadness mixed with the knowledge that everyone around you is still enjoying those once-special holidays can be very upsetting. Serina acknowledges all of these negative holiday transformations that are born of loss, but she also shares ways that she has begun to move forward. One suggestion she has is to make new traditions. Holding onto the past, resolutely unable to move forward, can immortalize the pain and prevent any healing that could happen during those holidays. Serina suggests using new traditions as a way to remember and move forward; as she says, “it won’t be the same and it never will be, but start a tradition that brings the memory of your loved one back to the holiday season.”
By Pam Crowley Andrews, Executive Director, Firefly Fund and Allison Rosen, 3D Communications for Firefly Fund
Congratulations to Denise Sousa, Becky McGuire, and Daniel Worley, our top 3 winners! Thank you to all who participated.
Kaila Guy, ASMD
We are looking for your help! Connecting with others by sharing YOUR story is how our community can support one another. Will you share your story on what NNPDF means to you? These stories will be included in our upcoming newsletters, shared on our social pages and with some of our partners. Send your stories, along with a photo to 
“Pfrieger’s Digest” is written by Frank Pfrieger of 
The Family Journeys Blog posts have been updated for the month of November. Follow our 4 NNPDF families from across the country as their share monthly updates to bring some light and positivity to our community! Our blogger families share personal, uplifting, hopeful stories to show what it means to be living with Niemann-Pick disease. 
Advice From a Niemann-Pick disease Mom: Avoid Google Taylor Sabky explains how she came to learn about this devastating disease. 
Advocating for ASMD: A Rare Disease Story Taylor Sabky shares her journey in learning about ASMD. 
To Help and Be Helped Taylor Sabky shares how helping can bring healing. 
Firefly Fund Chat: NNPDF Pam and Chris Andrews have a conversation with NNPDF Executive Director Joslyn Crowe and Board Chair Justin Hopkin about their journeys to NNPDF, the resources NNPDF provides, and what is on the horizon. 
Teacher’s mission: Defeat disease that cost her son his life By sharing Purnell’s story, Taylor Sabky puts a human side to ASMD. 
Thank you to all who joined for the ASMD Clinical Update webinar. If you were unable to join us or would like to watch again, the recording is now available. 
HEY SUPERHEROES!
Last month we were hoping to share some exciting news about recruitment of a US-based Clinical Research Associate as part of a project backed by the Ara Parseghian Medical Research Fund, so we are delighted to welcome Lakeisha Simmons to role.
Now is your chance to get awesome NNPDF gear!
Doctor, Parent, and Rare Disease Advocate Dr. Justin Hopkin, NNPDF Board Chair, shares his family’s’ story with Patient Worthy. 
Invisible Threads: Uncovering the Purpose of Community Connections For Isabelle and Garrett’s families, connecting to others living with a rare disease offered answers, courage and hope. 
NNPDF’s Executive Director, Joslyn Crowe, spoke to CheckRare about How Families Cope with a New Rare Disease Diagnosis. 
Living Beyond My Rare Disease Evren Ayik descibes life living with ASMD in a recent article published by The Mighty. 
Why Awareness Days Matter to Me as a Parent of a Kid With a Rare Disease Meghann Ferguson, NNPDF Board member, spoke to The Mighty about why Niemann-Pick Awareness month matters. 
Thank you to Global Genes for raising awareness of Niemann-Pick disease with this Rare Leaders Report profile feature article of NNPDF Executive Director, Joslyn Crowe. 
Welcome to our new Family Journeys Blog! Follow 4 NNPDF families from across the country as their share monthly updates to brings some light and positivity to our community! Our blogger families share personal, uplifting, hopeful stories to show what it means to be living with Niemann-Pick disease and the brighter sides within our community. 
Sanofi Genzyme recognized Niemann-Pick Awareness Month with a special, global event focused on ASMD for internal colleagues on October 15. Sandy Cowie (President, INPDA) and Clara Fortune (US patient) shared their stories and educated over 800 Sanofi Genzyme team members from around the world about their experience with ASMD, signs, symptoms, and the challenges of living with such a disease so rare that you rarely meet someone else with your diagnosis.
NNPDF is continuously working to meet the needs of our community. An area of need that we hope to help support are those surrounding loss (bereavement) and loss related to the Niemann Pick Disease Diagnosis. NNPDF Family Services is offering a new direct services program to provide a confidential space for families to process the complexities related to the realities of loss on multiple levels.
As October is Niemann-Pick Awareness Month, we’ve been running a campaign across our social media channels to celebrate our achievements, which of course are your achievements! We have focused on how much the Registry has grown, not only in the volume of data but also in our role as a ground-breaking, patient disease registry.
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: We have received the following letter from Mallinckrodt. 
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.
In recognition of Newborn Screening Awareness Month we are sharing the Firefly Fund’s September Firefly Chat, which is sharing a recent discussion from the recent World Orphan Drug Congress from August, 2020. You can 
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.
We are pleased to announce Becky McGuire as NNPDF Board Chair. Becky’s cousin, 
Gita Naseri, PhD
Steven Erwood
Did you know that since 1993 NNPDF has funded 52 grants and 18 fellowships totaling over $4,000,000? NNPDF’s contributions to Niemann-Pick research 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.
Justin Hopkin Wears a Lot of Hats and Makes Them All Fit to Help Serve Others | NNPDF Board Chair was recently featured in Uplifting Athletes, a national nonprofit organization aligning college football with rare diseases. Justin, you are in inspiration to many and we are honored to have you serving our community. 
NNPDF Executive Director, Joslyn Crowe’s recent article was featured in Rare Disease Report on HCP Live. Read “Getting a Treatment Is Not the End of the Road in Rare Diseases” 
We’re proud to share NNPDF Executive Director, Joslyn Crowe’s, podcast episode on Global Genes RARECast where she speaks about how NNPDF is preparing the patient community for the availability of the first therapies for niemann-pick disease, the issues people will need to consider, and how families may think differently about their futures. 
Sanofi Genzyme recently announced the launch of 
Thank you to all who participated in the NNPDF’s Challenges of Access Survey. The data collected will assess challenges you have experienced in accessing care, coverage for medications, services, and devices, and will be a valuable tool for us in advocating for Niemann-Pick families to ensure coverage for FDA-approved treatments. The data is being analyzed now and we will share the results in early 2021.
Interview with Christopher Sousa
Do you remember being told when Connor was diagnosed or learning about the diagnosis?
How does Niemann-Pick affect your daily life, for you personally versus your family as a unit?
What advice would you give to other young people in your situation when they first learn of their sibling’s diagnosis?
April Clemenza
When did you receive your diagnosis? What led you to diagnosis?
What were the first steps your parents took after diagnosis?
How did you learn about NNPDF?
How has being an NNPDF member benefitted your family?
Welcome to our new Family Journeys Blog! Follow 4 NNPDF families from across the country as their share monthly updates to brings some light and positivity to our community! Our blogger families share personal, uplifting, hopeful stories to show what it means to be living with Niemann-Pick disease and the brighter sides within our community. 
MARK YOUR CALENDARS! The 2021 NNPDF – INPDA Global Family Support & Medical Conference will be held July 29 – August 2, 2021 in Orlando, Florida at the Wyndham Grand Orlando Resort Bonnet Creek. Registration and hotel information to follow.
Dear Friends,
Congratulations to Evren Ayik who will receive Sanofi Genzyme’s TORCH Award on August 27th. The event will be held on Facebook Live – to join Sanofi Genzyme for the 2020 virtual TORCH Awards on August 27th from 7 – 8pm EST 
NNPDF Board Member, Meghann Ferguson, along with her son Liam (NPC), were recently interviewed by CBS-affiliate television channel WDMV. They shared the importance of more awareness of NPC and the urgent need for approved treatments. For complete story 
During the Sanofi Genzyme Patient Advocacy Leadership Summit, NNPDF Executive Director Joslyn Crowe shared her insights on “Developing Your Board & Growing Your Organization” and the “Do’s & Don’ts of Going Virtual”.
NNPDF Executive Director, Joslyn Crowe, was featured as an expert speaker at the Inborn Errors of Metabolism Drug Development Summit speaking on “Effective Collaborations Between Industry and Patient Organizations”. She also spoke on the panel discussion “Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need” and as a workshop leader on “Patient Access & Recruitment for Rare & Ultra-Rare Metabolic Diseases”.
Coming this fall, NNPDF will hold support sessions for living with loss. These 8-week programs will be led a trained professional and will offer a small-group space to share, learn, and heal. The program will be held virtually and will require an 8-week commitment.
PALS is flying again! The decision to pause their missions due to COVID-19 was a very hard one to make. Now after careful consideration, PALS is beginning to resume select missions again! 
The NNPDF Community app is now live! Use the Community section of the app to keep you connected to NNPDF friends & families and to stay up to date on our latest news – Download it today to your smartphone or tablet!
RTI Health Solutions is recruiting certain types of patients with Niemann-Pick ASMD, type A/B or type B. 
Firefly Fund’s NPC Newborn Screening Initiative had the opportunity to deliver a presentation at this year’s NNPDF Family conference, updating participants on the latest activities with this important community activity. We focused on two research initiatives: 1) the ScreenPlus pilot newborn screening study is making final preparations to launch in the state of New York, amidst the covid-19 pandemic; and 2) the NPC Sibling Study, being conducted in collaboration with a research entity called RDMD, to compare health outcomes of NPC-affected siblings based on timing of initiation of treatment. You can check out the presentation 
Woodcliff Lake School District Fundraiser for NNPDF
We quickly added more family services program content to our weekly schedule to meet the growing needs of our families. Small group discussions have been held by Zoom to provide a space for families to gather, share, and decompress.
Registration is free of charge for all families. Our virtual conference will be interactive and engaging with the latest in clinical updates, educational sessions from top speakers, an important keynote address from the U.S. Food and Drug Administration (FDA), plus much more!
The NNPDF Conference & Community app is now live! Download it today to your smartphone or desktop/laptop! This interactive tool will be used during the Family Support & Medical Conference and we’ll continue to add programming and content throughout the year.
Cora Sterling
What caused you to get involved in the Niemann-Pick community? How did you learn about NNPDF?
What has prompted you to continue volunteering the NNPDF Conference?
Patient handbooks are available to NPC patients and their families. This fillable electronic booklet is a place for you to record and print out important health and information to have available in case of an emergency. 
Sanofi Genzyme COVID-19 Study FAQ: In response to questions we have received from you about challenges faced in trial participation during COVID19, Sanofi Genzyme has provided the following information. 
Jamberry Niemann-Pick disease Nail Wraps
We have all experienced a new normal over the past couple of months as we adjust to various stay at home orders across the country. We’ve been apart due to social distancing, but by catching up through our NNPDF Community Connections Chats and Coffee and, of course, during Storytime with Cora, we’ve been able to spend time together every week! Special thanks to everyone that has been able to join us. Whether it was connecting during coffee time or sharing experiences during one of the NPC or ASMD chats, we have enjoyed seeing you and getting to know you better! Haven’t had the chance to make one of the chats? Make sure to 
Help Needed for Niemann Pick Disease Type A & B (NPD A & B) / ASMD Research
Conan Donnelly
Author C.S. Lewis, when musing on the death of his wife, described grief as “a sort of invisible blanket between the world and me.” For most, the invisible blanket Lewis describes eventually is lifted; for others, it grows ever heavier. Grief, by its very definition, is a severe emotion felt deeply and broadly. In the face of tragedy or loss, it is expected to feel devastated and overwhelmed.