Novartis seeks to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington’s disease (HD)

Drug remains under study for SMA but Novartis said it wants to see if trials in humans can duplicate preclinical models in which branaplam was shown to reduce levels of a mutant protein that plays role in HD, for which there are no approved disease modifying therapies.

Source:

Reuters Health