News

Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths
Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
Researchers reduce cardiac arrhythmia after heart attacks using gene therapy
During a severe heart attack many heart muscle cells die and are replaced by scar tissue to stabilize the heart wall.
Boston Herald1mon
Second death reported with gene therapy for muscular dystrophy
Elevidys is the first gene therapy approved in the U.S. for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. Related Articles Today in History ...
CNN1y
FDA expands approval of first gene therapy for rare form of ... - CNN
The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...
New Atlas1y
Muscular dystrophy-reversing gene therapy: Human trials in 2 years
A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage.
Reuters6mon
Novartis gene therapy helps children with rare muscle disorder in study ...
Novartis was testing the therapy, onasemnogene abeparvovec or OAV101 IT, in a late-stage trial of patients between the ages of 2 and less than 18 with spinal muscular atrophy (SMA). Sign up here.
Science Daily2mon
Duchenne muscular dystrophy gene therapy | ScienceDaily
Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus. Neurology , 2025; 104 (11) DOI: 10.1212/WNL.0000000000213604 Cite This Page : ...
The American Journal of Managed Care5mon
SGT-003 Gene Therapy Shows Potential in DMD
SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...
News-Medical.Net on MSN10d
New UAB-developed gene therapy for ALS receives orphan drug designation
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...
Healio3mon
Gene therapy improves functional outcomes for boys with ... - Healio
SAN DIEGO — Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory boys with Duchenne muscular dystrophy, interim data show ...