Brain tumor, breast cancer, colon cancer, congenital heart disease, heart arrhythmia. See more conditions.
(for at least one location)
Describes the nature of a clinical study. Types include:
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. NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. 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.
Study statuses change often. Please contact us for help.
Open for enrollment
The purposes of this study are to establish the natural history of the 3 diseases including their characteristics, management and outcomes; to assess clinical effectiveness of management and quality of care; to provide an inventory of patients for recruitment to interventional studies; to establish genotype-phenotype correlations; and to support post marketing obligations for approved therapies.
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