Genetic and environmental risk factors for advanced alcoholic liver disease (ALD) and nonalcoholic fatty liver disease (NAFLD) seem likely to include factors that in uence the severity of ...

I want his name to live on, and I want us to do some good for him.” “That’s a life that saved so many, and will continue to ...

Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C. Wainwright. Sangamo plans to use pivotal Phase I/II data to build an accelerated ...

Adrenoleukodystrophy is a debilitating x-linked disease caused by mutations in the ABCD1 gene. Developments in the clinical and basic science aspects of this disease ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported ...

PORTLAND — Twenty-seven states and the District of Columbia on Monday filed a lawsuit in bankruptcy court seeking to block the sale of personal genetic data by 23andMe without customer consent.

NEXUS (NCT04528706), a 96-week, phase 2/3, open-label, multicentre study conducted between February 13, 2020 and April 2025, enrolled boys aged 2–12 years with X-linked adrenoleukodystrophy with white ...

Maybe she’s born with it, maybe it’s … genetic optimization? Prospective parents using in vitro fertilization (IVF) will soon be able to select embryos based on their potential risk for ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and speed of new drug approvals.

Nine-month-old KJ Muldoon is the first patient to successfully receive personalized CRISPR gene editing therapy. After 300 days in the hospital, baby KJ has returned home.