We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”Īt WMS 2021, data from the ongoing open label RAINBOWFISH study were presented. “Our goal is to continue to lead the way in developing transformative medicines for neuromuscular diseases.

Also, the data from SRP-9001 have helped to optimise the design of the upcoming Phase III trial for DMD,” said Levi Garraway, M.D., Ph.D, Roche’s Chief Medical Officer and Head of Global Product Development.

“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients. The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to six weeks of age and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).

Further presentations included data from studies supporting the efficacy, safety, and durability of gene therapy, SRP-9001, in the treatment of Duchenne muscular dystrophy (DMD)īasel, 24 September 2021 - Roche (SIX: RO, ROG OTCQX: RHHBY) today announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20 – 24 September 2021.

Evrysdi has demonstrated consistent clinically meaningful efficacy in adults, children, and babies two months and older and is now approved in 58 countries worldwide.

New data show pre-symptomatic babies with spinal muscular atrophy (SMA) treated with Evrysdi maintained the ability to swallow.

Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders

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