FDA Approves First Treatment for Niemann-Pick Disease, Type C | FDA For Immediate Release:September 20, 2024 Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in …
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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 …
7th Annual DART To The Finish Charity Walk * Sat. * Sept. 28, 2024
Gather your friends and family and join is for another invigorating 2-mile walk with beautiful views of Long Island Sound around Tod’s Point in Old Greenwich, and help us raise much needed funds to find a cure for Niemann-Pick type C disease. No beach pass, no problem. DART has you covered! The morning always makes …