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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.

United statesKylie okeefeStuartw peltzJane bajToddler developmentDrug administrationTherapeutics incAmerican academy of neurologyRoche groupVirtual annualChief executive officerMotor scaleBayley scalesPhiladelphia infant testHammersmith infant neurological examinationSpinal muscular atrophy

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

United statesNathalie meetzLevi garrawayDaniel grotzkyPatrick barthBasil darrasKarsten kleineToddler developmentEuropean commissionDrug administrationHead of global product developmentProfessor of neurology at harvard medical schoolToddler development third editionMuscular atrophy program at boston children hospitalAmerican academy of neurologyRoche group

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

United statesNathalie meetzLevi garrawayDaniel grotzkyJon kaspar bayardKarl mahlerSabine borngrBasil darrasGerard tobinBruno eschliBirgit masjostLisa tuomiLoren kalmPatrick barthKarsten kleineToddler development

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™
prnewswire.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from prnewswire.com Daily Mail and Mail on Sunday newspapers.

United statesKylie okeefeStuartw peltzJane bajToddler developmentDrug administrationTherapeutics incAmerican academy of neurologyRoche groupVirtual annualChief executive officerMotor scaleBayley scalesPhiladelphia infant testHammersmith infant neurological examinationSpinal muscular atrophy

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

Roche s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA)
pharmiweb.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from pharmiweb.com Daily Mail and Mail on Sunday newspapers.

United statesLevi garrawayBasil darrasToddler developmentEuropean commissionDrug administrationHead of global product developmentProfessor of neurology at harvard medical schoolToddler development third editionMuscular atrophy program at boston children hospitalAmerican academy of neurologyRoche groupPharmaceuticals industryAmerican academyAnnual meetingHarvard medical school