Risdiplam
Risdiplam is an experimental drug being developed by F. Hoffmann-La Roche, PTC Therapeutics and SMA Foundation to treat spinal muscular atrophy. It appears to be effective orally and is a small molecule. It is a pyridazine derivative that works by increasing the amount of functional survival of motor neuron protein produced by the SMN2 gene through modifying its splicing pattern.
Risdiplam has shown good safety and efficacy in phase II–III clinical trials., Roche has applied for marketing authorisation in Brazil, Chile, Indonesia, Russia, South Korea, Taiwan and the United States while applications in China and the European Union are to be submitted by mid-2020.
Since late 2019, Roche has been offering the drug globally for free for eligible people through an expanded access programme.
