The first infant to undergo gene-editing therapy has accomplished another first — his first steps. KJ Muldoon is walking and ...

An Illinois family pushed for both boys to receive the potentially life changing medical treatment after one was approved and ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

Aradigm Health, a new benefits platform aimed at easing the financial sting of coverage for cell and gene therapies, has launched out of stealth. | Aradigm Health, new benefits platform aimed at ...

At the Cleveland Clinic, doctors are using a one-time gene-editing therapy that alters a patient’s own blood-forming stem cells to correct the genetic mutation responsible for the disease. Physicians ...

A miracle baby who was treated with a pioneering gene-therapy at CHOP has reached a major milestone as he took his first ...

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...