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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

- Tertiary endpoint data show trends of continuous improvement in activities of daily living, fatigue, and endurance over 24 months - These data indic ....

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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. ....

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

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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 ....

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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 S ....

United States , Nathalie Meetz , Levi Garraway , Daniel Grotzky , Jon Kaspar Bayard , Karl Mahler , Sabine Borngr , Basil Darras , Gerard Tobin , Bruno Eschli , Birgit Masjost , Lisa Tuomi , Loren Kalm , Patrick Barth , Karsten Kleine , Toddler Development , European Commission , Drug Administration , Head Of Global Product Development , Professor Of Neurology At Harvard Medical School , Toddler Development Third Edition , Muscular Atrophy Program At Boston Children Hospital , Roche Group , American Academy Of Neurology , Pharmaceuticals Industry , Roche Group Media Relations ,