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 ...

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

The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

Solid Bio said recently it has started dosing additional patients in its phase 1/2 trial of SGT-001 – called IGNITE DMD – and will have further results from the first three patients treated ...

In a medical first that could revolutionize treatment for thousands of rare genetic diseases, doctors have successfully used ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

After 3 doses of tailor-made treatment, an infant with a rare, life-threatening urea cycle disorder is thriving ...

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