HOW CAN WE HELP YOU TODAY?
- AveXis publishes encouraging interim results for Phase 1 study of gene therapy AVXS-101
- SMN Protein Can Be Measured in Blood – Implications for Clinical Trials
- Results from phase 1 clinical trial of Nusinersen in children published.
- Sign up for a 2016 Challenge Event!
WHY SUPPORT THE SMA TRUST?Knowing that The SMA Trust is actively funding and driving research is both a comfort and a necessity. A cure for this devastating disease is clearly the ultimate goal but in the meantime, any research which leads to enhanced quality of life for anyone suffering from SMA must be supported, as any parent who has lost a child to SMA will understand. - Hannah Razzell
SMA Type I, 19 December 2011 – 21 September 2012