A global network of non-profit organisations supporting those affected by Niemann-Pick diseases

Zevra NPC Community Letter – MIPLYFFA now available (USA)

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) •...

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...