Photos accompanying this announcement are available at: ...

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 ...

Company completed Type C meeting with FDA, aligning on its plan to submit an sNDA; submission expected around end of 1Q25. If approved, FILSPARI could be the first and only approv ...

Acute and chronic treatment for hepatic encephalopathy acquired from Mallinckrodt Pharmaceuticals in September 2024Extended pre-clinical, ...

Day One Bio aims to bring new cancer therapies to pediatric patients faster amid evolving financial incentives, such as the ...

The fireside chat will be webcasted and available under “Events & Presentations” in the Investor Relations section of the Company’s website at www.rhythmtx.com. A replay of the webcast will be ...

Measles and mumps are viral diseases so rare that fewer than one in 40,000 ... Mumps was a leading cause of deafness in children and rubella sickened thousands of pregnant women, causing many ...

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 ...