F.D.A. Faces Upset Over Denials of New Drugs

A recent New York Times article highlights ongoing discussions about how the FDA evaluates and approves treatments for rare diseases, and how federal health policy decisions could impact the pace of drug development.

For families affected by rare diseases, these conversations matter. When conditions are progressive and patient populations are small, timely access to promising therapies can make a meaningful difference in the lives of patients and their families.

https://www.nytimes.com/2026/03/05/health/fda-drugs-rare-diseases-rfk-jr.html

We are grateful to the Selser family for sharing their story and helping bring attention to the realities families face while waiting for new treatments. Personal stories like theirs help ensure that policymakers, researchers, and the public understand what is truly at stake.