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Niemann-Pick Diseases clinical trials

View clinical trials related to Niemann-Pick Diseases.

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NCT ID: NCT04799522 Available - Clinical trials for Sphingomyelin Lipidosis

Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

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

The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, during the period between completion of the clinical trials necessary to support submission of a request for marketing authorization and receipt of a final response from the applicable regulatory authority in the country where this program is available.

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.