These are exciting times, with fresh news about research developments appearing every week.
Find out about the latest research news below and also visit our Breaking News in Research page.
Joint letter to NICE regarding the suspension of the appraisal process for ridisplamRead more
Updates on Roche's SMA drug, risdiplam
An update on Zolgensma for SMA
PTC Therapeutics today announced data demonstrating the ongoing benefit of risdiplam for the treatment of all types of spinal muscular atrophy (SMA).
Muscular Dystrophy UK is investing £1.2 million into 9 new research projects this year, bringing our total research portfolio to over £10m.
The European Medicines Agency (EMA) has granted Orphan Drug designation to Cytokinetic's reldesemtiv for the treatment of patients with SMA.
The US FDA has granted approval to Zolgensma, the first gene therapy for SMA. Although the approval process in Europe is separate to the US, it’s a major step forward.
Scholar Rock has announced today that it has begun dosing patients in its Phase II clinical trial in Type 2 and Type 3 SMA.
The 71st annual meeting of the American Academy of Neurology, held this week, has seen the release of positive data from multiple SMA clinical trials.
The trial results so far show that children treated with Zolgensma are living longer and achieving significant development milestones.
The 12th UK Neuromuscular Translational Research Conference was held in Newcastle on 4-5 April 2019.