Miglustat improves swallowing in children and adolescents with Niemann-Pick type C1 disease.
Letter from the NTA to the FDA announcing the formation of the Accelerator
Dear Dr. Woodcock and Dr. Donohue: As leaders across the Neimann-Pick Type C (NPC) Community, we are writing to inform you of a significant collaboration among our organizations aligning efforts to help bring treatments to patients and families. The NPC Therapy Acceleration Initiative will ensure a strategic and coordinated approach to our shared objectives. The externally-led Patient-Focused Drug Development Meeting … Read More