Niemann-Pick Type C – Rare Disease Case Study: Surpassing Timelines & Rapid Enrolment In An Ultra-Rare Paediatric Study

The sponsor needed to produce a new batch of their product with concentrations adapted based on the weight of paediatric patients.

An observational study was needed to identify and profile patients before progressing to an interventional study. Patients enrolled were between 2 to 18 years of age with a diagnosis of NP-C.

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