International Niemann–Pick Disease Alliance
• AQNEURSA™ (ak-nur-sah) is the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC) •...
Read storyAugust 2nd, 2024 Dear NPC Community, Today, the Genetic Metabolic Diseases Advisory Committee (GeMDAC) met to...
Read storyAzafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. Study...
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