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Oral treatment for mucopolysaccharidosis VI: Outcomes of the first phase IIa study with odiparcil.
Guffon N, Chowdary P, Teles EL, Hughes D, Hennermann JB, Huot-Marchand P, Faudot-Vernier E, Lacombe O, Fiquet A, Richard MP, Abitbol JL, Tallandier M, Hendriksz CJ. Guffon N, et al. Among authors: faudot vernier e. J Inherit Metab Dis. 2022 Mar;45(2):340-352. doi: 10.1002/jimd.12467. Epub 2021 Dec 30. J Inherit Metab Dis. 2022. PMID: 34910312 Clinical Trial.