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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group. Schoser B, et al. Among authors: laforet p. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Lancet Neurol. 2021. PMID: 34800400 Clinical Trial.
Clinical features of late-onset Pompe disease: a prospective cohort study.
Wokke JH, Escolar DM, Pestronk A, Jaffe KM, Carter GT, van den Berg LH, Florence JM, Mayhew J, Skrinar A, Corzo D, Laforet P. Wokke JH, et al. Among authors: laforet p. Muscle Nerve. 2008 Oct;38(4):1236-45. doi: 10.1002/mus.21025. Muscle Nerve. 2008. PMID: 18816591
Rigid spine syndrome revealing late-onset Pompe disease.
Laforêt P, Doppler V, Caillaud C, Laloui K, Claeys KG, Richard P, Ferreiro A, Eymard B. Laforêt P, et al. Neuromuscul Disord. 2010 Feb;20(2):128-30. doi: 10.1016/j.nmd.2009.11.006. Epub 2009 Dec 14. Neuromuscul Disord. 2010. PMID: 20005713
A randomized study of alglucosidase alfa in late-onset Pompe's disease.
van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA. van der Ploeg AT, et al. Among authors: laforet p. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859. N Engl J Med. 2010. PMID: 20393176 Free article. Clinical Trial.
Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa.
van der Ploeg AT, Barohn R, Carlson L, Charrow J, Clemens PR, Hopkin RJ, Kishnani PS, Laforêt P, Morgan C, Nations S, Pestronk A, Plotkin H, Rosenbloom BE, Sims KB, Tsao E. van der Ploeg AT, et al. Among authors: laforet p. Mol Genet Metab. 2012 Nov;107(3):456-61. doi: 10.1016/j.ymgme.2012.09.015. Epub 2012 Sep 17. Mol Genet Metab. 2012. PMID: 23031366 Clinical Trial.
Polyglucosan body myopathy caused by defective ubiquitin ligase RBCK1.
Nilsson J, Schoser B, Laforet P, Kalev O, Lindberg C, Romero NB, Dávila López M, Akman HO, Wahbi K, Iglseder S, Eggers C, Engel AG, Dimauro S, Oldfors A. Nilsson J, et al. Among authors: laforet p. Ann Neurol. 2013 Dec;74(6):914-9. doi: 10.1002/ana.23963. Ann Neurol. 2013. PMID: 23798481 Free article.
339 results