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F Hoffmann-La Roche Ltd: New four year data for Roche s Evrysdi reinforce long-term efficacy and safety profile in some of the most severely affected people with types 2 and 3 spinal muscular atrophy (SMA)

Data from pivotal SUNFISH study showed increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease

United statesUnited kingdomLevi garrawaySabine borngrGerard tobinBruno eschliLaurent servaisBirgit masjostLoren kalmNathalie altermattMuscular dystrophy associationSociety for medicines researchToddler developmentBritish pharmacological societyDrug administrationHead of global product development

Scholar Rock Reports Full Year 2022 Financial Results and Highlights Business Progress

CAMBRIDGE, Mass. (BUSINESS WIRE) Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play

United kingdomFrance generalDennis riedlJay backstromTed mylesAriane lovellTraceym saccoRushmie nofsingerScholar rock incSociety for immunotherapy of cancerDrug administrationExchange commissionScholar rock holding corporationEuropean medicines agencyScholar rockChief executive officer

Nusinersen May Stabilize Disease Status in SMA Types 2 and 3

Treatment with nusinersen may decrease disease progression in pediatric patients who are nonambulatory with spinal muscular atrophy types 2 and 3.

Orphanet journalHammersmith functional motor scale expandedRevised upper limb module

Scholar Rock Reports Positive Trends in Phase 2 Study of Therapy for Non-ambulatory SMA2 and SMA3 Patients

Scholar Rock Reports Positive Trends in Phase 2 Study of Therapy for Non-ambulatory SMA2 and SMA3 Patients
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United statesComunidad autonoma de catalunaJay backstromInternational scientific congressEuropean medicines agencyDrug administrationRevised hammersmith scalePediatric evaluationDisability inventory computer adaptive testEndurance shuttle boxBlock testRevised upper limb moduleHammersmith functional motor scale expandedFast trackOrphan drugRare pediatric disease

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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United statesUnited kingdomComunidad autonoma de catalunaJay backstromAriane lovellRushmie nofsingerBasil darrasScholar rock incInternational scientific congressDrug administrationHarvard medical schoolExchange commissionEuropean medicines agencyAssociate neurologist in chiefBoston childrenChief executive officer