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In this issue of Neurology®, we are publishing an important article and accompanying editorial1,2 on the development of genetic methods that lay the groundwork for a clinical trial testing the effectiveness of genetic treatment for alpha-sarcoglycan (α-SG) deficiency (LGMD type 2D). This genetic disorder is responsible for one of the limb-girdle muscular dystrophies for which there is no treatment. However, the report that appears this week makes a case that genetic treatment may be feasible.
Some of our readers may wonder why a proof of concept article is important. To be sure, the most immediate impact of a finding of this type will be felt by researchers in the field. But in a very real sense, it is equally important for those of us who care for persons with muscular dystrophies, and it is certainly relevant for those who live with this disorder. The editorial team at Neurology® is committed to keeping our readership informed on issues related to developing treatments. We will update practitioners on future …
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