If you’ve landed on this page, your child or grandchild probably said a weird word you've never heard before in your life, ...

An athlete who plays power soccer, Luke says the challenges motivate him.

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted ...

Sheffield boy Kairo Barranco, seven, was diagnosed with Duchenne muscular dystrophy in September 2024.

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Researchers developed a gene therapy for LAMA2-related muscular dystrophy that restored muscle and nerve function in mice.

Dyne announced the results of new analyses of cardiac and pulmonary function amongst all DELIVER participants who were ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced presentations on SGT-003, its ...

In Trumbull County, a local nonprofit will once again be hiding colorful candy-filled eggs in yards this Easter to help a ...

Early diagnosis of rare diseases in children can significantly improve treatment outcomes and quality of life. Learn symptoms ...

Mr T, one of the most recognisable faces of 1980s pop culture, made a rare public appearance over the weekend. See photos.

Vinay Prasad’s exit — his second from the agency in less than a year — comes after sharp criticism of his handling of drug ...