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Research Studies

Metabolic Disease Program Research Studies

Find research studies available to children cared for by the Metabolic Disease Program team.

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Recruitment Status

Study categories

Eligible age

Arimoclomol Early Access Program for Niemann-Pick Disease

This study will provide early access to an investigational drug which does not yet have marketing authorization for human use, Arimoclomol, for a group of patients suffering from Niemann-Pick Disease Type C that are not eligible for or able to participate in other clinical trials.

Phase: Phase IV

Actively recruiting: Yes

Category: Adults, Children

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