May 2022 Newsletter
2022 Family Support & Medical Conference
NNPDF Cora Sterling Endurance Award | NNPDF Research Fellowship Applications
INPDA – ASMD Webinar Recording | ASMD Accelerate | Pfrieger’s Digest
Transport NPC | Clinical Trial Updates | Community News Updates | Surveys & Studies
Fundraising | Membership | Emergency Hardship Program
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 Deadlines
- Conference Registration by June 24th to receive your complimentary 2022 Family Conference shirt
- Hotel Reservations by June 27th to receive the NNPDF hotel group rate
- Online Registration by July 22nd on site registration will be available
DEADLINES APPROACHING!
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.
NNPDF Research Fellowship Applications
NNPDF is accepting Research Fellowship Applications. The Peter G. Pentchev 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. Many former grantees and fellows have remained active contributors in the Niemann-Pick Disease space. Contact nnpdf@nnpdf.org with questions. Application deadline is June 1st.
INPDA - ASMD Webinar Recording Available
We are pleased to share the collaborative webinar recording entitled The impacts of olipudase alfa on pediatric patients with ASMD and their families: Results of an international survey. The webinar was hosted by the INPDA, INPDR, NNPDF, and NPUK.
Thank you to those that joined us, and to our featured speakers; Sandy Cowie, President, International Niemann-Pick Disease Alliance (INPDA), Conan Donnelly, CEO, International Niemann-Pick Disease Registry (INPDR), and Justin Hopkin, Chair, National Niemann-Pick Disease Foundation (NNPDF).
Join ASMD Accelerate, Help Change the Future of ASMD Care
Just over a year ago, Wylder Nation, NNPDF, and PicnicHealth launched ASMD Accelerate to help support the ASMD research community. Thank you so much to the 15 families who have contributed their child’s de-identified medical data in support of ASMD research! Every child’s story can make a difference for the future of ASMD care.
If you are interested in joining ASMD Accelerate and making a difference in the future of ASMD research, we are still enrolling new members! This research study is a way your child’s journey can contribute to ASMD research without the need for travel, lab testing, or physical record collection coordination. Additionally, if your child’s journey with ASMD has ended this study can help carry on their legacy and contribute to improving the future of ASMD care.
Visit picnichealth.com/asmd-wylder-nation or email asmdstudy@picnichealth.com with any questions.
Join ASMD Accelerate.
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.
Transport NPC
Cyclo Therapeutics’ May newsletter is live and shares a new column with you called “Powerful Patient Advocates.” This month features an interview with Barbara Lazarus. Read the newsletter.
Clinical Trial Update
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 Orphazyme: Orphazyme announces update on in-court restructuring proceedings. NNPDF has received the following press release which speaks to the progress the team has made in finding a potential path forward for arimoclomol. Read the press release.
Community News Update
Update from Orphazyme:
Orphazyme to sell to KemPharm, Inc.: NNPDF has received the following news from Orphazyme: Orphazyme A/S under In-Court-Restructuring to sell substantially all of its assets and business activities to KemPharm, Inc.
Read Orphazyme press release.
Read KemPharm press release.
We will continue to keep you informed as more information is available. If you have any questions, please contact Joslyn Crowe, NNPDF Executive Director, at jcrowe@nnpdf.org.
Update from Cyclo Therapeutics:
Cyclo Therapeutics has shared the following letter to the NPC community. 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 familyservices@nnpdf.org for any questions.
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 Claudia Rae and Allison Reiter 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 familyservices@nnpdf.org 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 familyservices@nnpdf.org if you have any questions about this program.
