A global network of non-profit organisations supporting those affected by Niemann-Pick diseases.

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Zevra Therapeutics Announces Publication of Four-Year Real-World Data from U.S. Early Access Program Evaluating MIPLYFFA® (arimoclomol) in Niemann-Pick Disease Type C (NPC)

Zevra Therapeutics Announces Publication of Positive MIPLYFFA® Data from Pediatric Substudy in Infants with Niemann-Pick Disease Type C (NPC)

Global Milestone Reached in Niemann-Pick Type C Clinical Research

Advancing Research for the Global NPC Community The International Niemann-Pick Disease Alliance (INPDA) is pleased to share an important...