Case Studies
Orphan Drug Designation for Niemann-Pick Disease
This project seeks to obtain orphan drug designation for a new treatment for Niemann-Pick Disease, a rare and fatal genetic disorder.
Gene Therapy for Alkaptonuria
This project aims to develop gene therapy treatments for Alkaptonuria through collaboration between researchers and patient advocacy groups.
Biomarker Development for Gaucher Disease
This project aims to develop novel biomarkers for Gaucher Disease to improve diagnosis and monitoring.