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

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ASMD Project – The Invisible Years – Survey

Proposed Patient Organisation Recruitment Communication At Niemann-Pick UK, we know that the road to an ASMD diagnosis is often...

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)