Patient and Caregiver Outcomes after Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey.
Advances in therapy（2023）
关键词
Caregiver impact,Gene replacement therapy,Health care resource utilization,Motor milestones,Onasemnogene abeparvovec,Health-related quality of life,Spinal muscular atrophy,Survey,Unmet needs
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