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
Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™
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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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April 12, 2021 Taysha Gene Therapies has acquired exclusive worldwide rights to a clinical-stage adeno-associated virus 9 (AAV9) gene therapy program (TSHA-120) for the treatment of giant axonal neuropathy.
TSHA-120 is currently being evaluated in an ongoing open-label, nonrandomized, dose-escalation clinical trial conducted by the U.S. National Institute of Neurological Disorders and Stroke (NINDS) division of the National Institutes of Health (NIH). The primary end point is safety, and a primary measure of TSHA-120 s clinical efficacy is the Motor Function Measure 32 (MFM32) score. TSHA-120 has demonstrated the potential to improve MFM32 scores in patients. Additional data from the study are expected later this year.