The trial was small — just 12 children were treated in one or both ears — but it offers important lessons for researchers ...
“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...
For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...
Opal Sandy, now 3, underwent a gene therapy injection to her right ear in 2023. Top admiral steps down abruptly amid drug ...
AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the publication in peer-reviewed journal Nature Medicine of 12-month ...
New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial participants.
A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...
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 ...
The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the life-threatening bleeds caused by the condition.
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
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