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) • Approval follows positive Phase III data demonstrating significant improvements in neurological symptoms and functional benefits that could be seen within...
August 2nd, 2024 Dear NPC Community, Today, the Genetic Metabolic Diseases Advisory Committee (GeMDAC) met to review Zevra Therapeutics’ arimoclomol as an orally delivered treatment for Niemann-Pick disease type C (NPC). The committee, an independent panel...
Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C. Study primary endpoint met, with confirmed safety profile Investigators and Patient Organisations supportive of continued development Leiden, The Netherlands, 19 June...
IntraBio Inc is pleased to announce that The New England Journal of Medicine (NEJM) has published a Science Behind the Study Expert Perspective Editorial: N-Acetyl-l-Leucine and Neurodegenerative Disease. The Editorial reports on the broad potential of N-acetyl-L-leucine (IB1001) as...
IntraBio Inc is pleased to announce that The New England Journal of Medicine (NEJM) has published the detailed results of the Phase 3, Pivotal Trial with N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). New England...