Therapeutic advances in spinal muscular atrophy: a review of clinical, safety, and economic considerations. (PubMed, Front Pharmacol) - Sep 30, 2026 - "In this comprehensive narrative review, we extensively summarise the clinical efficacy and real-world effectiveness of approved disease-modifying therapies, including nusinersen, onasemnogene abeparvovec, and risdiplam, underscoring significant improvements in survival, motor milestones, and quality of life, particularly with early treatment initiation...Lessons from SMA provide a valuable framework for rare-disease management, highlighting the importance of mechanistic insights, early intervention, collaborative research, patient advocacy, and innovative health technology assessment to improve access and outcomes. Our aim is for the review to guide clinical practice, research priorities, and health policy by synthesising contemporary advances and challenges in SMA treatment and care."
HEOR • Journal • Review • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMA4 • SMN1
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Elvira Meni Maria Gkrinia; Yun Wah Lam
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