Consensus on gene therapy for spinal muscular atrophy in Taiwan. (PubMed, J Formos Med Assoc) - Sep 17, 2026 - "The advent of three FDA-approved disease-modifying therapies-onasemnogene abeparvovec, nusinersen, and risdiplam-has markedly improved therapeutic prospects...This consensus recommends incorporating SMA into the newborn screening program for early diagnosis and prompt treatment, and emphasizes that gene therapy should be evaluated based on SMN2 copy number and clinical condition. Presymptomatic treatment is critical for optimal motor outcomes, and multidisciplinary care teams are essential for comprehensive long-term management."
Journal • Review • CNS Disorders • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Pediatrics • Rare Diseases • SMA4 • SMN1 • SMN2
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Che-Sheng Ho; Fuu-Jen Tsai; I-Ching Chou; I-Jun Chou; Inn-Chi Lee; Kuang-Lin Lin; Li-Ping Tsai; Ming-Yuh Chang; Shuan-Pei Lin; Shyi-Jou Chen; Ting-Rong Hsu; Wang-Tso Lee; Wen-Chen Liang; Wen-Chin Weng; Yin-Hsiu Chien; Yuh-Jyh Jong; Yung-Ting Kuo
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