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Pick Disease of the Brain clinical trials

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NCT ID: NCT04316637 Available - Clinical trials for Niemann-Pick Disease, Type C

Early Access Program With Arimoclomol in US Patients With NPC

Start date: n/a
Phase:
Study type: Expanded Access

NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.