An oral presentation at ASCO & EHA Congress from the CARTITUDE-1 study highlighting heavily pre-treated patients that remain alive and progression-free for more than 5 years after a single CARVYKTI® ...

With grim prognosis hanging overhead, doctors and scientists at universities and institutions across the U.S. worked ...

A CRISPR treatment seems to have been effective for a baby’s devastating disease, but it is not clear whether such bespoke ...

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

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

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 ...

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

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

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

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

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