Economic Burden of Sanfilippo Syndrome in the United States
Preprint in Research Square (November 2023)
The most recent citing publications are shown below. View all 46 publications that cite this research output on Dimensions.
Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scores
Article in Orphanet Journal of Rare Diseases (October 2023)
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
Article in Molecular Therapy (October 2023)