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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

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

United statesComunidad autonoma de catalunaJay backstromBasil darrasScholar rock incInternational scientific congressDrug administrationHarvard medical schoolExchange commissionEuropean medicines agencyAssociate neurologist in chiefBoston childrenChief executive officerPediatric evaluationDisability inventory computer adaptive testReported outcome measurement information system