International Niemann-Pick Disease Alliance (INPDA)

IntraBio Announces U.S. FDA Approval of AQNEURSA for the Treatment of Niemann- Pick Disease Type C

• AQNEURSA™ (ak-nur-sah) is the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC) •...
Read story
GeMDAC Votes Favorably on Arimoclomol for NPC: Key Step Toward FDA Approval

August 2nd, 2024 Dear NPC Community, Today, the Genetic Metabolic Diseases Advisory Committee (GeMDAC) met to...
Read story
Azafaros announces positive topline Phase 2 study data with nizubaglustat

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. Study...
Read story

View all events