Twice-Weekly Outpatient Rehabilitation Intervention for Young Children With Spinal Muscular Atrophy Treated With Genetic-Based Therapies: Protocol for a Feasibility Study
BackgroundSpinal muscular atrophy (SMA) is a progressive neuromuscular disorder that causes muscle weakness and is the leading genetic cause of infant mortality worldwide. While no definitive cure exists, the approval of 3 genetic-based therapies in Canada since 2018 has led...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
JMIR Publications
2023-11-01
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Series: | JMIR Research Protocols |
Online Access: | https://www.researchprotocols.org/2023/1/e46363 |