October 12, 2017
Genentech/Roche has provided the below community statement with clinical trial updates for SUNFISH (Type 2/3). The first patient is now enrolled in SUNFISH, as the trial advances to part two, which is designed to be a pivotal marketing registration trial.
September 28, 2017
To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , “Spinraza and Advanced Therapies: a stakeholder special,” covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected by SMA,...
September 19, 2017
Novartis has released the following update for the LMI070 (branaplam) clinical trial, on behalf of the program team.
Dear SMA community,
For years, our researchers have been working hard to develop a new treatment for SMA. One promising compound in our neuroscience...