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

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 ...

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

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

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

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 ...

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

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

Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults ...

AMD Phase 3 timing, Otsuka funding to 2028, and mixed 4D-710 data with financial risk. See why FDMT stock is a hold.

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

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