A girl born without a brain celebrates her 20th birthday, reversing medical predictions that her chances of survival are slim ...

Preclinical studies demonstrated significant efficacy on systemic inflammation and organ function in ACLF disease modelsSafety data from Phase 1 on healthy volunteers and initial efficacy signals from ...

A dream has come true for one Rantoul family after their son, Ezra, got his Make-A-Wish granted this week. Two-year-old Ezra ...

Seles’ first symptoms appeared suddenly around three years ago. She began having double vision and weakness in her arms and ...

Elafibranor (Iqirvo) was well tolerated and led to sustained improvements in liver enzyme levels and itch severity through 28 weeks in patients with primary sclerosing cholangitis (PSC), according to ...

Federal health officials are investigating an outbreak of a rare infant disease in 10 states linked to baby formula that was ...

A once-rare disease that causes a person to develop a severe allergy to red meat is seeing an “explosive rise” across America ...

The Rhode Island Department of Health (RIDOH) is advising families and consumers that a case of infant botulism has been ...

Mia Hamant, a University of Washington soccer goalie, died from a rare form of kidney cancer on Nov. 6. She was 21.

A single infusion of CRISPR Therapeutics' experimental gene therapy was safe and reduced levels of harmful LDL cholesterol ...

A 15-year-old girl with a rare chromosome disorder received a complete room renovation Friday that her family says will ...

Iain Cheeseman and colleagues reveal the underappreciated role of single genes producing multiple proteins in atypical ...