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Niemann-Pick Disease News
FDA Approves Miplyffa for Treatment of Niemann-Pick Disease, Type C
MONDAY, Sept. 30, 2024 – The U.S. Food and Drug Administration has approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in...
FDA Approves Aqneursa (levacetylleucine) for the Treatment of Niemann-Pick Disease Type C
AUSTIN, TX, September 25, 2024– IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug A...
FDA Approves Miplyffa (arimoclomol) for the Treatment of Niemann-Pick Disease Type C
CELEBRATION, Fla., Sept. 20, 2024 (GLOBE NEWSWIRE) – Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that the ...