The U.S. Food and Drug Administration has declined to approve Scholar Rock's drug for a rare neuromuscular disease, citing ...

The seventh annual Calves to Cure DMD charity cattle auction raised funds to help fund research for Duchenne muscular dystrophy.

MLAB Biosciences, a biotechnology company pioneering novel therapies for neuromuscular disease, today announced the ...

In recent years, Roche has made a habit of oscillating between first and second place on rare disease patient groups’ annual ...

Switching off a single enzyme in immune cells protected mice from obesity, type 2 diabetes, and fatty liver disease in a new ...

World Heart Day raises awareness about cardiovascular diseases, prevention strategies, and the importance of heart health ...

Kinea Bio Inc., a biotechnology company dedicated to developing transformative genetic medicines, today announced two major ...

A kickball tournament this weekend at the Potsdam Moose Lodge, 6907 Route 56, will raise money for medical expenses for a 1-year-old Norwood girl being treated for a rare neurological disorder. Brynn ...

Then came the devastating news: The Drurys’ only child had a rare, terminal disease so aggressive, most kids don’t live to ...

Increasing the cholesterol-lowering protein ApoM in mice improved retinal health and may be the key to slowing age-related ...

By improving gait stability, Ionis’ zilganersen could be “potentially disease modifying,” according to analysts at William ...

Lorna Rothery spoke with Victoria Hedley, co-lead of the Rare Disease Research UK Hub, about initiatives aimed at advancing ...