Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...
New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
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Experimental gene therapy restores immune system function in children with genetic immune disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...
News Medical on MSN
Gene therapy shows long-term success in children with rare immune disorder
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...
An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder, a promising ...
In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...
A novel, validated seven-gene transcriptomic signature can predict TNF inhibitor response in patients with rheumatoid ...
A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using ‘longevity genes’ ...
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