On November 5, 2026, the New England Journal of Medicine published a study on the long-term efficacy of AAV8-mediated gene therapy (GNT0003) in ...
The health, safety, and well-being of people living with spinal muscular atrophy (SMA) and their families are always our ...
Cryoport, Inc. (NASDAQ: CYRX) ("Cryoport" or the "Company"), a leading global provider of integrated temperature-controlled ...
Novartis has acknowledged seven previously unknown instances in which tumors were found in children who were treated with its ...
Italian nonprofit Fondazione Telethon and US nonprofit Orphan Therapeutics Accelerator are collaborating to bring curative ...
On RGX-202 (Duchenne), Simpson reiterated the regulatory timeline: "Preparations are well underway to submit the BLA under the accelerated approval pathway in the first quarter of 2027, supporting ...
Advocates are sounding the alarm on social media as they learn from their doctors that Novartis has received reports of ...
Three years ago, a trial for a sickle cell gene-editing therapy a decade in the making ended abruptly after the first patient ...
In a first-in-human clinical trial, a gene therapy was found to improve swallowing ability for people with oculopharyngeal ...
Health and Me on MSN
Drug-resistant epileptic seizures reduce by over 90% with experimental gene therapy
In a recent trial involving three patients with drug-resistant epilepsy, researchers combined gene therapy and an ...
Rocket Pharmaceuticals is honored with the 2026 BioTech Breakthrough award for its innovation in gene therapy for LAD-I.
Nanoscope Therapeutics, a biotechnology company developing gene therapies for retinal degenerative diseases, has appointed Damon Daulerio as Executive Director of... Read More ...
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