Genoskin

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

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

This project involves conducting a Health Technology Assessment (HTA) for new treatments targeting Mucopolysaccharidosis, a group of rare metabolic disorders.

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.