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  • Spinal muscular atrophy: a ...
    Tizzano, Eduardo F; Finkel, Richard S

    Neuromuscular disorders : NMD, 10/2017, Letnik: 27, Številka: 10
    Journal Article

    Highlights • SMA has evolving phenotypes due to improved standard of care and new treatments • The clinician should be alert to identify new patterns of motor development in SMA • Other organ systems may develop signs of dysfunction as these patients live longer • Early diagnosis, including newborn screening, warrants consideration • Strategies targeting the CNS and peripheral tissues are warrant consideration