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Phase 4 nusinersen trial underway in infants, children with spinal muscular atrophy

from Pharmacy Times

Phase 4 nusinersen trial underway in infants, children with spinal muscular atrophy Pharmacy Times A phase 4 study is underway to assess the safety of nusinersen in infants and children with spinal muscular atrophy who have unmet clinical needs following treatment with onasemnogene abeparvovec. The study, known as RESPOND, aims to evaluate the safety and efficacy of nusinersen in patients with suboptimal responses to onasemnogene abeparvovec. Individuals living with SMA do not produce enough survival motor neurons, which support sitting, walking, or basic functions of life such as breathing and swallowing. READ MORE

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