Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
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DOI: 10.1002/acn3.51519
Abstract: To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA). read more here.
Keywords: safety; muscular atrophy; abeparvovec spinal; onasemnogene abeparvovec ... See more keywords