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.
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.
The core study was a 26-week, randomized, double-blind, placebo-controlled trial in patients receiving ERT and an open-label, noncomparative, single-dose cohort not receiving ERT. ...Of 20 patients enrolled, 13 (65.0%) completed the study. Odiparcil increased total …
The core study was a 26-week, randomized, double-blind, placebo-controlled trial in patients receiving ERT and an open-label, …