U.S. Food and Drug Administration approved Alnylam's drug to treat a rare and deadly heart disease on Thursday, allowing the ...

Ever wondered what it’d be like to win millions in a Mr Beast competition? Jeff Allen did exactly that. He reveals ...

The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...

A study in Science Translational Medicine found when patients take the drug nitisinone, their blood becomes deadly to ...

Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...

Machine learning and unwavering support helped Joseph Coates overcome POEMS syndrome. Discover how computational medicine is ...

He took the stage and was great last week at the Grand Ole Opry’s 100th anniversary show, and now he is down to just two ...

Cerebrocostomandibular syndrome is a rare genetic disorder characterized by a combination of abnormalities affecting the ...

Author LJ Smith, who was behind the Vampire Diaries series that inspired the hit TV show of the same name, has died at the ...

The startup is using CRISPR tools to stop errant expression of a gene linked to facioscapulohumeral muscular dystrophy, a ...

Low doses of a drug already approved for treating rare genetic conditions in humans kills mosquitoes dead. Scientists say it ...

The Delhi High Court has observed that availability of a drug for treatment of rare diseases at economical and competitive ...