New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)
TreatSMA are extremely happy to announce that Lianne Harris has joined the team as a volunteer to assist with Fundraising and Events Co-ordination.
We are now listed as a charity on Amazon Smile. By choosing us as your selected charity Amazon will donate 0.5% of the net purchase price (excluding VAT, returns and shipping fees) of eligible purchases to us. You can set…
Novartis has made the decision to discontinue development of branaplam, an investigational oral, once-weekly RNA splicing modulator, for the treatment of spinal muscular atrophy (SMA). This was a difficult decision that was made as the result of rapid advancements in the SMA treatment landscape in recent years.
We received news yesterday that 241 people are now enrolled on the EAMS for Risdiplam.