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Evrysdi™ Approved in Japan for the Treatment of Spinal Muscular Atrophy

Evrysdi™ Approved in Japan for the Treatment of Spinal Muscular Atrophy
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Pre-symptomatic Infants with Spinal Muscular Atrophy Achieved Same Motor Milestones as Healthy Children After Treatment with Evrysdi™ in RAINBOWFISH

New Roche data for Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with spinal muscular atrophy (SMA)

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new interim data from two studies of Evrysdi® (risdiplam); JEWELFISH and RAINBOWFISH. Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another .

Genentech s Evrysdi Continues to Improve Motor Function and Survival in Babies With Type 1 Spinal Muscular Atrophy (SMA)

Genentech s Evrysdi Continues to Improve Motor Function and Survival in Babies With Type 1 Spinal Muscular Atrophy (SMA) - Evrysdi increased survival and reduced need for permanent ventilation - - Evrysdi has proven efficacy across adults, children and babies 2 months and older - Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. The study also showed Evrysdi continued to improve survival, improve ability to feed orally and reduce the need for permanent ventilation . Exploratory data suggested Evrysdi continued to improve the ability to swallow and reduce hospitalizations compared to the natural course of Type 1 SMA. Safety for E

Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™

Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™ - Evrysdi increased survival and reduced need for permanent ventilation - News provided by Share this article Share this article SOUTH PLAINFIELD, N.J., April 15, 2021 /PRNewswire/  PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that results from Part 2 of the pivotal FIREFISH trial demonstrated that infants with type 1 spinal muscular atrophy (SMA) treated with Evrysdi™ (risdiplam) obtained increases in survival and sustained improvements in achieving key motor milestones, including head control, sitting, rolling over, and further developing towards acquiring the ability to stand, and walk. Data were presented at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting.

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