Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Orphanet J Rare Dis. 2017 Jun 26;12(1):117. DOI:10.1186/s13023-017-0675-4

Ghosh ARust S,  Bigger BW, Jones SA, et al

https://www.ncbi.nlm.nih.gov/pubmed/28651568

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