Elania is one of 59 children born with ADA-SCID who have been cured using this gene therapy, which will soon be submitted for ...
Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...
These results are what we hoped for when we first began developing this approach,' one researcher said. View on euronews ...
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...
New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...
Stevie Ringel is the founder of the Kizuna Foundation, an organization seeking a treatment for a genetic retinal mutation ...
With a new COO and several consultants that were formerly as Spark Therapeutics, CorriXR plans on "rewriting the rules of ...
In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
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