TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...

GOMEKLI™ is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adult and pediatric ...

Three-year-old Hailey Wetmore was born with a rare birth defect. It’s called cloacal exstrophy and it impacts her excretory functions. “At first, we were told that Hailey may not live,” her mother ...

The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...

To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1‐800-FDA-1088 or .

Hirschsprung disease is a rare condition affecting one in every 5,000 babies, according to the National Institutes of Health.

MMR vaccines are safe and effective, but fewer children are getting them.

A good Samaritan called an audible and gave an 8-year-old battling a rare chronic disease the opportunity of a lifetime.

The path to rare disease product innovation involves interdependencies between clinical, regulatory, and commercial. Understanding these and the challenges and considerations along the way is ...

Disney+ remains the go-to platform for streaming the best kids movies. Thanks to the streamer’s mix of Disney vault classics and modern hits, the little ones in the family will be satisfied with ...

However, certain diseases and medical conditions continue to receive less funding and attention compared to others. Patients with rare medical conditions and diseases are often part of a ...

Sophie Hessekiel is a Newsweek writer based in Los Angeles. Her focus is on celebrity news and entertainment. She has also written extensively about culture and relationships for a variety of ...