Arimoclomol Early Access Program for Niemann-Pick Disease

Arimoclomol Early Access Program for Niemann-Pick Disease

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This study is now recruiting. Learn more about enrolling here.

Description
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.

What to expect
The study will involve the following procedures: (1) Administration of study drug (Arimoclomol)(2) Blood tests and urine tests(3) Medical history interviews(4) Questionnaire completion(5) Physical exams/height/weight(6) Review of medical records.

Related specialties
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