Join Us in Making a Difference
Upcoming Events
Together, let's make a meaningful impact and bring hope to those facing Niemann-Pick Disease Type C.
Help us make a difference in the childhood dementia fight for equitable access to research and treatment.
We're putting these flyers up at local business during Easter week.
Please let us know if you'd like a laminted flyer to hang at a business you know or own.
June 8-11, 2024
The annual conference will include sessions on NPC1 structure and function, autophagy, cholesterol trafficking, lysosomal biology, therapeutics, gene therapy, biomarkers, and newborn screening. There will also be multiple breakout sessions.
*Please note that this is an invite only conference. If you wish to be added to the invite list please contact Sean Kassen at skassen@nd.edu.
NNPDF Family support & Medical Conference
July 11-13, 2024
Location: Salt Lake City, Marriot City Salt Lake City, Utah
NORD® LIVING RARE, LIVING STRONGER® PATIENT AND FAMILY FORUM
- June 7 - June 8 2024
RARE IMPACT AWARDS®
- June 8, 2024
Location: The Hilton Los Angeles/Universal City Hotel 555 Universal Hollywood Dr, Universal City, CA, United States
Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation
- Noah Stansfield
Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.
August 22, 2023