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 science, and strong leadership within government agencies. This ambitious claim...
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 disease. This therapy targets patients suffering from familial chylomicronemia syndrome...
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, researchers, and others.“To encourage robust, good-faith discussion about issues raised...
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 comes as he aims to provide more support and resources...
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 one of the heart's lower chambers does not fully develop....
BREAKING NEWS
Reforming FDA Advisory Committee Policy for Conflict of Interest-Free Decisions
Peter J. Pitts, a distinguished figure in the realm of healthcare regulation, recently shared his insights on the critical issue of FDA Advisory Committee...
