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UnknownScientific rationale for a higher dose of nusinersen.
Not available
S-EPMC9186144 | biostudies-literature
UnknownPersonalising Outcomes after Child Cardiac Arrest (POCCA): design and recruitment challenges of a multicentre, observational study.
Not available
S-EPMC7592297 | biostudies-literature
UnknownDEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results.
Not available
S-EPMC10578235 | biostudies-literature
UnknownImpact of economic factors, social health and stressful life events on physical health-related quality of life trajectories in older Australians.
Not available
S-EPMC8532095 | biostudies-literature
UnknownEfficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
Not available
S-EPMC7505441 | biostudies-literature
UnknownNusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.
Not available
S-EPMC7127286 | biostudies-literature
UnknownPhase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.
Not available
S-EPMC6218284 | biostudies-literature
UnknownVamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.
Not available
S-EPMC7011869 | biostudies-literature
UnknownCirculating protein biomarkers identified in two independent clinical trial cohorts of glucocorticoid-naive Duchenne muscular dystrophy patients.
Not available
S-EPMC12618936 | biostudies-literature
UnknownProspective cohort study of spinal muscular atrophy types 2 and 3.
Not available
S-EPMC3525313 | biostudies-literature
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