Spinal muscular atrophy as a blueprint for precision therapy in neuromuscular disease. (PubMed, Expert Rev Mol Med) - Sep 24, 2026 - "The evolution of SMA therapies has transformed neurogenetics. Clinical benchmarks have successfully shifted from reactive, symptomatic management to proactive, molecularly targeted precision medicine."
Journal • Review • CNS Disorders • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMA4 • SMN1 • SMN2
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Busra Cesur Ergun; Busra Cetin; Ezgi Erbasan; Melike Aliciaslan; Salih Sanlioglu
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