Fig. 2From: A master protocol to investigate a novel therapy acetyl-l-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasiaExtension phase schema. Patients will be assessed approximately 4 times over a 64-week period: at the start of the extension phase, after 6 months of treatment, 1 year of treatment, and after a 42-day (+ 14 day) post-extension-phase treatment washoutBack to article page