Medana

This project seeks to obtain orphan drug designation for a new treatment for Niemann-Pick Disease, a rare and fatal genetic disorder.

This project focuses on designing clinical trials for Fibrodysplasia Ossificans Progressiva, a rare and debilitating condition that causes soft tissues to turn into bone.

This project aims to develop gene therapy treatments for Alkaptonuria through collaboration between researchers and patient advocacy groups.

This project aims to develop novel biomarkers for Gaucher Disease to improve diagnosis and monitoring.