Outcomes of combination therapy with nusinersen, onasemnogene abeparvovec, and risdiplam over 3.5 years in a patient with prenatally diagnosed spinal muscular atrophy type 0: A case report
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Background: Recently introduced disease-modifying therapies have markedly improved survival and motor outcomes, particularly when initiated presymptomatically or in the early symptomatic stage. However, reports on treatment of th... ...
