As communicated by Roche, the first site to run the Rainbowfish clinical trial had been activated and is ready to accept presymptomatic babies diagnosed with spinal muscular atrophy.
Genetic Alliance UK are working with other charities like TreatSMA on a policy project to improve access to medicines for rare conditions.
Recruitment in the first clinical trial of branaplam is complete.
Last week also at Myology 2019 conference Roche shared early observations from Sunfish Part I clinical trial of risdiplam.
We are excited to share early observations from Firefish Part I clinical trial of risdiplam. The data were shared with researchers at the Myology Congress in Bordeaux last week.
Another country is joininig the group of those that treat spinal musculatr atrophy as the health authorities in Argentina have decided to provide nusinersen (Spinraza) under national healthcare.