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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: hennermann jb. 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.
Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis.
Harmatz P, Cattaneo F, Ardigò D, Geraci S, Hennermann JB, Guffon N, Lund A, Hendriksz CJ, Borgwardt L. Harmatz P, et al. Among authors: hennermann jb. Mol Genet Metab. 2018 Jun;124(2):152-160. doi: 10.1016/j.ymgme.2018.04.003. Epub 2018 Apr 18. Mol Genet Metab. 2018. PMID: 29716835 Free article.
Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis.
Phillips D, Hennermann JB, Tylki-Szymanska A, Borgwardt L, Gil-Campos M, Guffon N, Amraoui Y, Geraci S, Ardigò D, Cattaneo F, Lund AM. Phillips D, et al. Among authors: hennermann jb. Mol Genet Metab Rep. 2020 Apr 8;23:100586. doi: 10.1016/j.ymgmr.2020.100586. eCollection 2020 Jun. Mol Genet Metab Rep. 2020. PMID: 32292699 Free PMC article.
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Diaz-Manera J, et al. Lancet Neurol. 2021 Dec;20(12):1012-1026. doi: 10.1016/S1474-4422(21)00241-6. Lancet Neurol. 2021. PMID: 34800399 Clinical Trial.
112 results