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It is currently the only approved gene therapy for Duchenne, available to ambulatory individuals aged 4 and older under an updated FDA label issued at the end of 2025
Kumakura Y, Vernaleken I, Buchholz HG, Borghammer P, Danielsen E, Grnder G, et al
Jako pierwsz namy na kilka minut na wosy mask zawierajc w skadzie keratyn
Prior to the availability of enzyme-replacement therapy, individuals with LAL-D were treated with lipid lowering medication, liver transplant, and stem cell transplant, none of which corrected the multisystem nature of the disorder
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