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

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

In April, the Deparment of Health and Human Services shuttered the committe that oversaw newborn screenings. Advocates hope ...

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

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

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

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

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

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

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