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New Advancement Toward The First Primary Endpoint Qualification Of A Digital Endpoint For Duchenne

Current methods for assessing ambulatory function in patients with Duchenne muscular dystrophy do not fully reflect how they experience DMD in their.

Region flamandeBruxelles capitaleFrance generalRobert debrRoxana donisa dreghiciLaurent servaisCarl morrisDonisa dreghiciHead of clinical developmentLouvain medical schoolNovo nordiskFranklin pierce collegeUniversity of medicineDepartment of physiologyEuropean medicines agencyInstitute of myology

New Four-Year Data for Genentech 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) Seite 1

20.03.2023 - Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new long-term data for Evrysdi (risdiplam) in a broad range of people aged 2-25 years with spinal muscular atrophy (SMA) from the pivotal SUNFISH study. Data . Seite 1

Laurent servaisLevi garrawayMuscular dystrophy associationRoche groupGlobal product developmentOxford neuromuscular centreScientific conferencePaediatric neuromuscular diseasesMotor functionRevised upper limb moduleGlobal productRoche holding

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

Solid Biosciences Presents New SGT-001 IGNITE DMD Study Results at World Muscle Society 2022 Congress Demonstrating Improvements in Ambulatory Function

Solid Biosciences Presents New SGT-001 IGNITE DMD Study Results at World Muscle Society 2022 Congress Demonstrating Improvements in Ambulatory Function
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Laurent servaisCaitlin lowiesolid biosciencesRoxana donisa dreghiciExchange commissionInternational hybrid annual congressUniversity of oxfordHead of clinical development at solidEuropean medicines agencySolid biosciences incWorld muscle societyNova scotiaPaediatric neuromuscular diseasesDystrophy association clinicalScientific conferenceSenior vice presidentClinical development

Solid Biosciences (SLDB) Presents New SGT-001 IGNITE DMD Study Results

Solid Biosciences (SLDB) Presents New SGT-001 IGNITE DMD Study Results
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Roxana donisa dreghiciLaurent servaisInternational hybrid annual congressUniversity of oxfordHead of clinical development at solidEuropean medicines agencyWorld muscle societyNova scotiaPaediatric neuromuscular diseasesDystrophy association clinicalScientific conferenceSenior vice presidentClinical development