Majority of newborn babies with spinal muscular atrophy (SMA) treated with Roche ’s Evrysdi able to sit independently after 1 year of treatment

RAINBOWFISH study met its primary endpoint with 80% of babies sitting without support for at least five seconds after 1 year of Evrysdi treatment – without treatment these babies would never be able to sitAll babies were able to swallow and feed orally and none required permanent ventilationEvrysdi is the only non-invasive SMA therapy and is approved in over 100 countries with more than 11,000 patients treated globallyBasel, 04 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi ® (risdiplam) in babies with pre-symptomatic SMA (n=26), aged from birth to six weeks. The data were presented at the 28th World Muscle Society (WMS) Congress, 3-7 October 2023.“Evrysdi is the only non-invasive SMA treatment and can be used within hours of birth, potentially allowing these babies to sit, stand and walk, similar to healthy individuals,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “Evrysdi h as now demonstrated its safety and efficacy in babies, children and adults and these compelling data continue to reinforce our confidence in this treatment.’’Clinical studies show that the loss of motor neurons may begin before symptoms start so initiating treatment early is critical for better outcomes. The RAINBOWFISH study included babies with two or more copies of theSMN2gene. Generally, t...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news