Neurogene said it would conduct daily monitoring for the "three Fs" in the week after dosing with the gene therapy.

After a patient died following a high dose of Neurogene’s Rett syndrome gene therapy candidate, the biotech is sharing ...

One week after he was born, KJ Muldoon was diagnosed with what doctors call a CPS-1 deficiency, essentially meaning he was ...

Treated with an individualized gene-editing therapy that corrects mutations directly on the genome, a patient shows ...

A n infant with a life-threatening and incurable genetic disease has become the first human to successfully receive a ...

The first patient to receive this gene therapy after it was FDA cleared is now living pain-free for the first time. Bike riding is fun now for 12-year-old Rahemeen Nabeel, who lives in Cherry Hill ...

"It's a momentous breakthrough for families facing this devastating disease," said Dr. Claire Booth, leader of the London trial site, according to EurekAlert. The new gene therapy works by modifying ...

Martin, 20, was the first patient in St. Louis and among the first across the country to receive a new gene therapy for sickle ... Martin nearly died when he was about 18 months old and fluid ...

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...