Two new studies by Strauss et al. demonstrated safe and effective pre-symptomatic delivery of gene therapy in children with spinal muscular atrophy (SMA). These results highlight the importance of newborn screening programs and early therapy delivery for SMA.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9418840 | PMC |
| http://dx.doi.org/10.1016/j.xcrm.2022.100725 | DOI Listing |
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