Since Elevidys' first approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory ...

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.

A range of other rare inherited diseases are now likely to be curable by similar procedures, the research team said ...

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 ...