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New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA

New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
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New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA - Scholar Rock Holding (NASDAQ:SRRK) - Press Release

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

Roche s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA)

Share: More than twice as many babies (61% vs. 29%) were able to sit without support for at least five seconds after 24 months compared to 12 months of treatment Evrysdi increased survival and reduced need for permanent ventilation Evrysdi has proven efficacy across adults, children and babies 2 months and older Basel, 15 April 2021 - Roche ((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 hospitalisations compared to the natural course

F Hoffmann-La Roche Ltd: Roche s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA)

F. Hoffmann-La Roche Ltd: Roche 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 Basel, 15 April 2021 - Roche (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 hospitalisations compared to the natural course of Type 1 SMA. Safety f

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