Spinal muscular atrophy is one of the most common inherited neuromuscular conditions; our understanding of the genetic pathology and translational research coming from this insight has made significant progress over the past decade. This short review provides the background of the disease along with the bench to bedside progress of some promising treatment options to develop better understanding of the present state of the disease.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7337203 | PMC |
| http://dx.doi.org/10.2147/DNND.S48420 | DOI Listing |
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