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 ...
New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...
“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...
News-Medical.Net on MSN
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 ...
In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...
In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...
MedPage Today on MSN
Gene Therapy Restores Hearing for Rare Cause of Congenital Deafness
In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...
Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...
Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...
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