Tag: Rare Diseases

President Donald Trump has the potential to revolutionize the landscape of rare disease treatments and save countless American lives through strategic policy changes, innovative...

Ionis Pharmaceuticals has achieved a significant milestone by obtaining FDA approval for their groundbreaking therapy, Tryngolza, designed to combat a rare and fatal genetic...

First Opinion is STAT’s platform for interesting, illuminating, and provocative articles about the life sciences writ large, written by biotech insiders, health care workers,...

Colin Farrell, the well-known actor, is taking a step forward in raising awareness for a rare genetic condition known as Angelman syndrome. This move...

Single ventricle heart disease is a rare condition that affects about 1 in 15,000 babies born in the U.S. This congenital defect occurs when...