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Niemann-Pick Disease Support Group

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Our support group for Niemann-Pick Disease has 1 questions and 3 members. Updated 5 Jun 2025.

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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…

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