Billy Joel’s daughter, Alexa Ray Joel, has revealed the steps he’s taking to improve his physical and mental health after he ...

The FDA has approved Denali Therapeutics’ enzyme replacement therapy for a genetic lysosomal storage disease, breaking the agency’s recent string of rejections for rare disease treatments. In ...

Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

Billy Joel's daughter Alexa Ray shares a health update on the "Piano Man's" battle with normal pressure hydrocephalus, ...

In brain tissue from people who died with aFTLD-U – a rare subtype of dementia marked by early behavior change – chromosome ...

Billy Joel's daughter Alexa Ray gives hopeful health update as he battles rare brain disorder - The singer, 76, said he ...

The Food and Drug Administration on Tuesday approved a generic medication for a rare brain disorder, while walking back ...

A Heber Springs woman is fighting an unimaginable battle, a rare brain disease that’s slowly taking her life.

OREGON, USA — Nearly two decades after first being identified, a rare and life-threatening brain-inflammation disease may soon have new treatment candidates. The disorder, called anti-NMDA ...

In a bid to better understand, and potentially treat, a host of conditions that affect early cognition, neurodevelopment and ...