Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

After sweeping cuts to US federal health agencies, the elimination of a committee responsible for recommending newborn ...

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Groundbreaking gene therapy offers hope for a boy with fatal muscular dystrophy, potentially slowing disease progression and ...

And for now, Logan and his family are reeling in the moments, one fish at a time.

Quince Therapeutics advances Ataxia-Telangiectasia treatment with innovative AIDE platform, insider support, and NEAT Phase 3 ...

Parents of seven-year-old boy are calling for a drug which improves muscle repair to be made available here as it is in some ...

Chronic illness affects 40 percent of school-aged children. Learn how caregivers can help reduce the stress and depression ...

A Tucson preschool teacher spotted a developmental quirk in a little boy who was then able to get early treatment for ...

Over the last two decades, the 41-year-old Chilean has spent her days bedridden, suffering from degenerative muscular dystrophy. When she finally loses her ability to speak or her lungs fail ...

Some see the shuttering of the Advisory Committee on Heritable Disorders in Newborns and Children as an opportunity for ...