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 ...

JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

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 ...

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

FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.

In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a ...

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

Gene therapy is now being used for certain cancers and sickle cell anemia.A South Florida teen was among the first to get ...

Findings from two clinical trials suggest Novartis' Itvisma is safe and effective for SMA patients over two years old.

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

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...