Guidance on Gene Replacement Therapy in Spinal Muscular Atrophy: A Canadian Perspective
- PMID: 34082851
- DOI: 10.1017/cjn.2021.125
Guidance on Gene Replacement Therapy in Spinal Muscular Atrophy: A Canadian Perspective
Keywords: Gene replacement therapy; Onasemnogene; Spinal muscular atrophy.
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