That leads to a buildup of ammonia that destroys the liver and can cause neurological damage with lifelong consequences. His ...

A recent report from the St James Health Department has brought renewed attention to the troubling reality of rodent ...

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

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

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

KJ received the first dose through an infusion in February, followed by two additional doses in March and April. Doctors ...

A team of doctors and scientists has used a tailor-made gene-editing therapy to treat an infant with a rare genetic condition ...

Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a ...

Breakthrough therapy may pave the way for personalized cures and a cheaper path to treating ultra-rare conditions.

“This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which ...

He was a star at the University of Tennessee when he began using LSD, mescaline and other drugs. He said he got clean while ...