Updates

Pfriegers Digest – 13th Issue

Pfriegers Digest – 13th Issue

Global Access Challenges for Niemann-Pick Therapies: Insights from an INPDA Community Survey

IntraBio Appoints Dr. Marc C. Patterson, US Chief Medical Officer

Update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses.

Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025

Pfriegers Digest – 12th Issue

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) • Approval follows positive Phase III data demonstrating significant improvements in neurological symptoms and functional benefits that could be seen within...

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 review Zevra Therapeutics’ arimoclomol as an orally delivered treatment for Niemann-Pick disease type C (NPC). The committee, an independent panel...