Early Access Program with Arimoclomol for the Treatment of Niemann-Pick disease Type C in the US

Brief description of study

In alignment with Orphazyme’s Early Access Policy, Orphazyme has launched a Single Patient Early Access Program (EAP) due to the urgent unmet medical need of patients with NPC and the fact that not all patients are able to participate in clinical trials for various reasons. In addition, Orphazyme will provide Early Access to arimoclomol on group level to bridge the time gap until the product becomes commercially available with this EAP.

Clinical Study Identifier: s20-01202

ClinicalTrials.gov Identifier: NCT04316637

Principal Investigator: Nicolas Julian Abreu.

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