Rare lysosomal disease registries: lessons learned over three decades of real-world evidence
Article in Orphanet Journal of Rare Diseases (October 2022)
The most recent citing publications are shown below. View all 23 publications that cite this research output on Dimensions.
Article in Orphanet Journal of Rare Diseases (October 2022)
Article in Expert Opinion on Drug Metabolism & Toxicology (May 2022)
Book chapter (November 2021)