The FDA’s new leadership must give rare disease patients a clearer path

At her confirmation hearing, Dr. Heidi Overton, President Donald Trump’s nominee to lead the Food and Drug Administration, ...

I've never felt so helpless: Pennsylvania mother shares baby's battle with rare disease

A central Pennsylvania baby is battling a rare genetic disease, and now her mother is sharing their journey in hopes of ...

A rare disease is taking his vision. A gene therapy could help — if he can get it

A well-connected family’s fight to access a potential treatment for their son highlights the barriers most rare disease patients struggle to overcome.
Zamin.uz

Five-year-old blogger who battled rare disease passes away

Five-year-old Brazilian social media star Alice Lima Carneiro has passed away after battling a rare aging syndrome. Along ...
BIOENGINEER

Six Experts Map the Future of Rare Metabolic Disease Research

Rare diseases are defined individually by their scarcity, but collectively they represent one of the largest burdens of unmet medical need in modern medicine. An estimated 300 million people worldwide ...
Ad Hoc News

Insmed Inc stock (US4576693075): pipeline update and growth story in rare diseases

Insmed Inc stock continues to attract attention from growth?oriented biotech investors as the company advances a portfolio of ...
Forbes

New $25 Million Accelerator Bets On Better Model For Rare Disease

This voice experience is generated by AI. Learn more. This voice experience is generated by AI. Learn more. A new Pittsburgh-based initiative, Rare Ventures, has launched with a $25 million commitment ...