A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Kavita Mehta suffers from beta thalassaemia, a genetic condition preventing her body from producing sufficient haemoglobin ...

The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

July 24 (Reuters) - Pfizer's (PFE.N), opens new tab gene therapy for hemophilia A significantly cut the number of annual bleeding episodes in patients with the rare disorder in a late-stage study and ...

Issued on behalf of Avant Technologies Inc. VANCOUVER, BC, Jan. 2, 2026 /CNW/ — Equity Insider News Commentary – The gene therapy market is surging toward $36.55 billion by ...

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, doctors said he's essentially "cured" thanks to gene therapy treatment at ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

Gene therapy may have the potential to cure Duchenne muscular dystrophy, but early results from clinical trials have not been as promising as researchers hoped. Research is ongoing. Duchenne muscular ...