A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act

por Fermaglich, Lewis J., Miller, Kathleen L.
Publicado 2023

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurolog...

por Miller, Kathleen L., Fermaglich, Lewis J., Maynard, Janet
Publicado 2021