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

Tevard Biosciences, Inc., a privately held biotechnology company pioneering tRNA-based therapies to cure a broad range of ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that ...

Health Secretary Neil Gray has been furiously condemned for failing to ensure access to a new muscular dystrophy drug for ...

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

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

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 ...

"The newer medications that are coming out, and a lot of them are approved, are much more focused on the problem," explains ...

Health Secretary Neil Gray has been furiously condemned for failing to ensure access to a new muscular dystrophy drug for desperate children. About 30 boys with a rare form of the killer disease ...

Earlier this year, Una Ennis took to social media with a heartfelt plea for funds so she could access specialist treatment abroad, following her seven-year-old son Archie’s muscular dystrophy ...