In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed and Health Law Scan, focusing on issues impacting the rare disease community.

Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most lack FDA-approved treatments. Developing therapies for ...

Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest and a visiting professor at the University of Paris School of Medicine. Over the summer, the ...

Almost by definition, rare diseases are so unusual information about them isn't readily accessible—not only for patients chasing a diagnosis but for the physicians trying to help them. HRA Pharma ...

A coalition of entrepreneurs, researchers, and biopharma industry advocates in suburban Westchester County, NY, is working to create a “hub” that would link them with partners in and outside the ...

Coming on the heels of an advisory committee in which the U.S. FDA and its independent advisers grappled with trying to fit an ultra-rare disease development program into the confines of the agency’s ...