Neuromuscular Disorders · 2021 · 19 citations · 11 references
MedicineNeurologyEarly DetectionDelayed DiagnosisSpinal Muscular AtrophyDelayed Diagnosis DetectionDetection DelayLate-onset Spinal Muscular AtrophyMuscle AtrophyPhysician Delay In DiagnosisSpinal Muscular Atrophy EvaluationSpinal Muscular Atrophy Screening
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Richard S. Finkel, Eugenio Mercuri, Basil T. Darras et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text
Richard S. Finkel, Eugenio Mercuri, Oscar H. Meyer et al. · Neuromuscular Disorders · 2017 · 667 citations · Full text