Ellie Van VelsenJan 83 minCommunityNP-C Corner: Will I be a lab rat? An exploration of clinical trials in the world of Niemann-Pick.Ellie explores what clinical trials are and how they impact patients of Niemann-Pick diseases.
ANPDFDec 28, 20231 minResearchZevra Therapeutics Media Release | December 27, 2023Zevra Therapeutics Announces Resubmission Of Arimoclomol New Drug Application To The U.S. Food And Drug Administration Read the full...
ANPDFDec 21, 20231 minResearchAzafaros Media Release | December 11, 2023Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled Read the full statement from...