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

Basel, 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. 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 investig ation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients. 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. “Our goal is to continue to lead the way in developing transformative medicines for neuromuscular diseases. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”At WMS 2021, data from the ongoing open label RAINBOWFISH study were presented. Four out of five of those treated with Evrysdi for at least 12 months achieved standing and walking independently within the World Health Organization windows for healthy children. In addition, all five babies maintained the ability to swallow and were able t...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news

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Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p
Source: Roche Media News - Category: Pharmaceuticals Source Type: news
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