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

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

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 ...

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

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics ...

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

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read more at straitstimes.com. Read more at straitstimes.com.

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 ...

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 ...

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

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, doctors said he's essentially "cured" thanks to gene therapy treatment at ...