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 …
Category
Showing: 1 - 3 of 38 RESULTS
Niemann-Pick Type C News
IntraBio files New Drug Application for N-acetyl-L-leucine (IB1001) for the treatment of NPC
IntraBio Inc. is pleased to share that the New Drug Application (NDA) for N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease type C (NPC) was submitted to the US Food and DrugAdministration (FDA) in January 2024. The FDA has a 60-day filing review period to determine whetherthe NDA is complete and accepted for review. With …
Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application to the FDA
December 27, 2023 at 7:30 AM EST CELEBRATION, Fla., Dec. 27, 2023 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC) to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based …