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 ...

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

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