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The Duchenne Lottery: Why Where a Child Lives May Now Matter More Than Ever
ELEVIDYS, the first Duchenne gene therapy, is approved in the US and Japan but rejected in Europe, leaving a child's access increasingly shaped by geography.
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#gene-therapy
All articles and blog posts tagged #gene-therapy.
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The DMD Research Pipeline: What's Beyond Approved Therapies
DMD research pipeline: CRISPR gene editing, next-generation gene therapy, exon skipping advances, anti-fibrotic approaches, and what families should know.
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Elevidys Explained: The First DMD Gene Therapy
Elevidys is the first FDA-approved gene therapy for Duchenne muscular dystrophy. What it is, who it is for, and what the regulatory record actually says.
Blog
Gene Therapy Duchenne Muscular Dystrophy Update
Gene therapy for Duchenne muscular dystrophy, updated: micro-dystrophin, FDA approval, safety signals, and the unresolved questions ahead.