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Spinal Muscular Atrophy as a Treatable Disease: Disease-Modifying Therapies Improving Clinical Outcomes and Quality of Life

Spinal muscular atrophy is one of the most common causes of infant mortality. Historically, SMA treatment consisted of supportive care only. The arrival of disease-modifying therapies has made a major impact on the prognosis for patients with SMA. This article highlights therapies approved by the FDA, such as survival motor neuron 2 (SMN2) modulators, SMN1 gene therapy, and the use of newborn screening to commence treatment in the pre-symptomatic period.
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