Revised Hammersmith Scale News Today : Breaking News, Live Updates & Top Stories | Vimarsana

Onasemnogene Abeparvovec Effective in Treatment of Older, Heavier Children with SMA

A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1.

United kingdomUnited statesSimon dannhauerLancet regional healthPhiladelphia infant testNeuromuscular disordersHammersmith infant neurological examinationRevised hammersmith scaleSpinal muscular atrophySma type 1Onasemnogene abeparvovec

Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial

Topline data from Phase 3 trial expected in Q4 2024 Company to present encore 36-month efficacy, safety, and patient-reported outcomes data from the Phase 2 TOPAZ trial extension at the 2023.

North charlestonSouth carolinaUnited statesJay backstromThomaso crawfordJohns hopkins universityWorld muscle societyCharleston area convention centerScholar rock incDrug administrationEuropean medicines agencyExchange commissionScholar rockAnnual congressChief executive officerRevised hammersmith scale

New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy

New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy
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United kingdomUnited statesRushmie nofsingerTeagan whiteDisney swanThomas crawfordJay backstromThomaso crawfordResearch clinical care meetingDolphin hotelsResearch clinical care meeting in orlandoDrug administrationScholar rock incExchange commissionEuropean medicines agencyJohns hopkins university

SMA in Adults: Interim Data Back Nusinersen Efficacy

New trial focuses on drug's performance in spinal muscular atrophy beyond childhood

University of rochesterNew yorkUnited statesJohn geverHealth indexNational institute of neurological disordersAmerican academy of neurologyRevised hammersmith scaleRevised upper limb moduleChad heatwoleAmerican academyNational instituteNeurological disordersManaging editor

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