People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

Pfizer (NYSE:PFE) is a global pharmaceutical giant that has long been a cornerstone of medical innovation and today boasts a ...

BOSTON, MA, April 10, 2025 (GLOBE NEWSWIRE) -- Odylia Therapeutics (Odylia) a nonprofit biotechnology company dedicated to expediting drug development for rare diseases, hosted a Rare Disease Day ...

A TIKTOKER has revealed that her 10-month-old daughter has died after suffering from a rare skin disease. In a heartbreaking social media clip, Hannah Campbell said her daughter Elliana died ...

Wilson's disease is a rare genetic disorder where the body cannot properly eliminate excess copper, causing it to build up in vital organs like the liver, brain, and eyes. Normally, copper is ...

That’s what the parents of a baby in Moore were devastated to hear when their little girl was born with a rare disease. But, this little girl far surpassed two—in fact, she’s about to ...

The therapeutic alliance between a patient and their physician — or the researcher studying their disease — is vital across all areas of human health. But in the field of rare diseases ...

Jalyn received a bone marrow transplant in December to address a rare, genetic condition. AeroAngel, a private jet service, coordinated a flight home for the family, as flying commercially could ...

According to Citeline, two-thirds of all drugs and biologics in the R&D portfolios of Amgen, Bristol Myers Squibb, Merck KGaA, Novartis, and Pfizer, for example, target rare diseases; and the ...

Although medical researchers have identified more than 7,000 rare diseases and made tremendous scientific breakthroughs in the past 20 years, more than 90% of rare diseases still lack any FDA ...

A medical breakthrough could result in the first treatment for rare but serious diseases in which genetic defects disrupt cellular energy production. Researchers at the University of Gothenburg ...