Campaign for Access to Treatments for Spinal Muscular Atrophy

The world may soon have a new, highly potent treatment for spinal muscular atrophy as Roche today announced that the key clinical trial of risdiplam has met its primary endpoint. Based on the trial data, which are also supported by TreatSMA’s own observations, risdiplam appears safe and highly effective in improving muscle function of children, adolescents and adults with SMA types 2 and 3 who have participated in the SUNFISH trial.

We are thrilled!

The US Food and Drug Administration has suspended all clinical trials of AVXS-101 (Zolgensma®) gene therapy administered intrathecally due to safety findings in animals.

On the 27th October at 7pm TreatSMA is hosting a live webinar alongside physio Marion Main. This will be a regular slot at the end of each month. It can be viewed on all our social media platforms live and…

As a part of our engagement with research into diet we came across this novel work and thought that general SMA population would be interested in the data.

Our Trustee Gennadiy Ilyashenko has been accepted as a patient organisation representative on the Spinraza managed access oversight committee. This is fantastic news.

Impressive! The results of NURTURE, a presymptomatic clinical trial of Spinraza, are out. The drug appears to prevent SMA altogether in the majority of children when started early!

A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children.