In the early days of his career, a young scientist named Emil Kakkis found himself deep in the world of...
The National MPS Society is pleased to share an update on RGX-121, an investigational gene therapy for MPS II...
After recent successful meetings with the FDA, Denali Therapeutics announced its plan to file for accelerated...
An Update on Allievex Dearest Sanfilippo Community Members and Allies, It is with deep...
Advocacy Groups Join Reagan-Udall Foundation and FDA for Workshop on Developing Biomarkers to Support...
