A five-year-old boy who received the world's most expensive drug as a baby has made "incredible progress" and can walk ...

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

Started in 2004 by a Duke University offensive lineman, Teamworks today says its software is used by more than 6,500 sports ...

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 ...

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

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), ...

DUBAI, United Arab Emirates--(BUSINESS WIRE)--Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a ...

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

The newly licensed intrathecal gene therapy expands life-changing treatment options for children and adults affected by ...

A baby born with a rare genetic disorder has taken his first steps after receiving a groundbreaking, personalized gene ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialized ...