University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

Nine people with a rare genetic condition that causes life-threatening inflammatory reactions appear to have been cured, after taking part in the first trial of a new version of a CRISPR-based gene ...

Hydrocephalus is a life-threatening condition that occurs in about 1 in 1,000 newborns and is often treated with invasive ...

Researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. Researchers at the Francis Crick Institute, UCL Great Ormond Street Institute ...

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic ...

A collaboration between researchers at New York University’s Grossman School of Medicine and Rocket Pharmaceuticals has produced preclinical evidence that gene replacement therapy successfully halts ...

(Reuters) -Novo Nordisk said on Monday the U.S. Food and Drug Administration (FDA) had approved its therapy to treat a rare genetic condition that affects the kidneys. The once-monthly injection, to ...

At Liv Hospital, specialists emphasize that stem cell therapy isn’t a one-size-fits-all solution. Instead, it is carefully indicated for specific diseases, patient profiles, and treatment ...

Researchers at the Francis Crick Institute, UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital for Children (GOSH) have designed a new genetic therapy that could ...

Oct 2 (Reuters) - Novo Nordisk (NOVOb.CO), opens new tab said on Monday the U.S. Food and Drug Administration (FDA) had approved its therapy to treat a rare genetic condition that affects the kidneys.

“Knowing there has been a huge step forward in the CMN research and there could be a chance of Ada’s CMN being reversed and possibly reducing Ada’s risk of developing melanoma, has blown our ...

Feb 12 (Reuters) - Takeda Pharmaceutical's (4502.T), opens new tab therapy for an allergic inflammation of the esophagus has received approval from the U.S. FDA, the Japanese drugmaker said on Monday, ...