VTS-270 IND Niemann-Pick Disease, Type C

Genetics

Protocol:VTS-270 IND

Condition or Disease: Niemann-Pick Disease, Type C

Study Type: Expanded Access

Status: Open for enrollment

Study Title: Intrathecal 2-Hydroxypropyl-ë_-cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1

Principal I investigator: Alan Ikeda, MD

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