Case Studies
Health Technology Assessment for Mucopolysaccharidosis (MPS)
This project involves conducting a Health Technology Assessment (HTA) for new treatments targeting Mucopolysaccharidosis, a group of rare metabolic disorders.
Biomarker Development for Gaucher Disease
This project aims to develop novel biomarkers for Gaucher Disease to improve diagnosis and monitoring.
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.