January 2024 Newsletter

Thank You to all that generously supported the NNPDF with your financial gifts in 2023, through donations, family fundraisers, and social media fundraisers. It is through the generosity of donors that we are able to provide vital unmet services to patients and families along their Niemann-Pick journey. Your support matters for our Niemann-Pick families and every dollar makes an impact. We can’t do it without you! Your ongoing commitment to our organization is greatly appreciated.

Rare Disease Day is February 29th!

Rare Disease Day is the globally-coordinated movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

Rare Disease Day promotes raising awareness and generating change for the 300 million people worldwide living with a rare disease such as Niemann-Pick, their families and caregivers. YOU can show your support on Rare Disease Day by raising awareness of Niemann-Pick Disease and what it means to be rare. Learn more and get involved!

Bringing Holiday Cheer Update

Thirty-seven 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 300 items for our families. Thank you again for providing much joy to our families.

Milestones

Congratulations to Alec Koujaian! Alec completed his 249th treatment and celebrated 10 years on Adrabetadex. Alec’s family feels blessed and remain hopeful for drug approval of Adrabetadex in 2024, along with the other NPC drugs looking for approval. Congratulations, Alec!

Abby Alvey is putting on her dancing shoes!! Abby is giving dance class another try after her lower leg botox and serial casting. She tried a little bit smaller of a class and she did amazing. Abby’s family is so proud that she never wants to give up. Thank you Central Virginia Dance Academy. Way to go Abby!

The Lazarus family celebrated 37 years with NPC warrior Daniel Lazarus on January 26th and wish for many more to come! Barb, Daniel’s Mom, expresses that every day is a gift, every year a blessing. Happy Birthday Daniel!!

Dee Reynolds recently celebrated the 36th anniversary of her 29th birthday… (do the math) by taking a cruise to the Caribbean! Happy Birthday Dee!

If you have a special Milestone you’d like to share with your Niemann-Pick community please send us a photo and the details to nnpdf@nnpdf.org.

NNPDF Store

With Rare Disease Day right around the corner did you know our store has some great items to help raise awareness for Niemann-Pick Disease? Awareness shirts are available in 3 designs with ASMD, NPC, or Niemann-Pick Disease options – available in T-Shirt or Hoodies. Personalization for your family is available.

Order today to get your gear by Rare Disease Day! Be sure to check out our NNPDF apparel and logo items and send us your photos with your new gear!

Allow 2 weeks for delivery. US only.

I Need A Cure for NPD - ASMD - Store Image

Someone I Love Needs a Cure - NPC - Store Image

Family Assistance & Support Program

The NNPDF Family Assistance & Support Program (FASP) provides relief to NNPDF U.S. member families that may be facing a financial obstacle that requires short-term financial assistance. Funding includes but is not limited to, specialized medical equipment and goods, utility bills, adaptive home repairs, home and car repairs, car payments, rent and mortgage payments, and bereavement expenses. Learn more about the NNPDF Family Assistance & Support Program.

Please contact Laurie Turner, Family Services Manager at 920-542-4038 or familyservices@nnpdf.org if you have any questions about this program.

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.

THANK YOU to Ashley Reel and Elsa Angulo who recently held Facebook Fundraisers!

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! Want to host your own fundraiser? Facebook Fundraisers are an easy option! Visit facebook.com/fund/NNPDF to get started!

ASMD Accelerate Now Enrolling All Forms of ASMD

Comprehensive Care Centers

NNPDF recently added Phoenix Children’s Hospital to our 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. Click here to learn more!

If you are connected with a medical team that would like to be added to the NNPDF Comprehensive Care Centers listing, please contact Laurie Turner at lturner@nnpdf.org.

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 Inc. is pleased to share that the New Drug Application (NDA) for N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease type C (NPC) was submitted to the US Food and Drug Administration (FDA) in January 2024. Read the complete update.

Update from Zevra Therapeutics: Zevra Therapeutics has announced the U.S. Food and Drug Administration’s (FDA) acceptance of the resubmission of the New Drug Application (NDA) for arimoclomol for treatment of Niemann-Pick disease type C. Read the complete announcement and Zevra’s message to the Niemann-Pick community below. Read the complete announcement and the message to the community.

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. Learn more.

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 familyservices@nnpdf.org or call 920-542-4038