International Niemann–Pick Disease Alliance

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 telangiectasia

We are pleased to share the following article “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 telangiectasia” which describes the collaborative development of an innovative master protocol and primary outcome assessment to investigate N-acetyl-L-leucine as a potential treatment for three rare disorders, including Niemann-Pick disease type C (NPC):

Fields_et_al-2021-Trials

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