Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the ...

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

Doctors will have to monitor the boy for years to ensure the experimental treatment is effective long-term. But for now, it's ...

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...

Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene ...

KJ Muldoon became the first patient to undergo personalized CRISPR treatment, a therapy that found the one uniquely mutated gene out of 20,000 in his little body, and fixed it.

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to ...

The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month ...

A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.