Tag: Rare Diseases
Reforming FDA to Help Rare Disease Patients
President Donald Trump has the potential to revolutionize the landscape of rare disease treatments and save countless American lives through strategic policy changes, innovative...
FDA Approval: Ionis Rare Disease Therapy Lowers Triglycerides
Ionis Pharmaceuticals has achieved a significant milestone by obtaining FDA approval for their groundbreaking therapy, Tryngolza, designed to combat a rare and fatal genetic...
Readers’ Opinions on Covid, Trump, Rare Diseases, and Institutional Neutrality: A Comprehensive Overview
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,...
Understanding Angelman Syndrome: What You Need to Know & Colin Farrell’s Son’s Battle
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...
Studying Rare Congenital Heart Defects: $13M Project Commits to Investigate Biological Mechanisms
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...
BREAKING NEWS
CDC Continues Publishing Papers Despite Firing Scientists: Flagship Journal Controversy
North Carolina Kids Find Lead in Applesauce: A Closer LookIn Hickory, North Carolina, the lead-poisoning-from-applesauce case started with two little kids showing concerning blood...
