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 ...
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 ...