July 9, 2024 at 9:00 AM EDT Meeting with the recently formed GeMDAC set for August 2, 2024 CELEBRATION, Fla., July 09, 2024 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has indicated that it will convene a meeting with the recently formed Genetic …
Azafaros announces positive topline Phase 2 study data with nizubaglustat in NPC and GM2
16 July 2024 Leiden, The Netherlands, 16 July 2024 – Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of either GM2 gangliosidosis or Niemann-Pick disease type C (NPC). About the Trial The trial was conducted across three sites in Brazil, …
Azafaros announces completion of Phase 2 RAINBOW study evaluating nizubaglustat in NPC and other rare disease patients
Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW (Phase 2 RAINBOW study NCT05758922). The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients older than 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the RAINBOW study is …