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Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy.
Fouarge E, Monseur A, Boulanger B, Annoussamy M, Seferian AM, De Lucia S, Lilien C, Thielemans L, Paradis K, Cowling BS, Freitag C, Carlin BP, Servais L; NatHis-MTM Study Group. Fouarge E, et al. Among authors: monseur a. Orphanet J Rare Dis. 2021 Jan 6;16(1):3. doi: 10.1186/s13023-020-01663-7. Orphanet J Rare Dis. 2021. PMID: 33407688 Free PMC article.
Myostatin: a Circulating Biomarker Correlating with Disease in Myotubular Myopathy Mice and Patients.
Koch C, Buono S, Menuet A, Robé A, Djeddi S, Kretz C, Gomez-Oca R, Depla M, Monseur A, Thielemans L, Servais L; NatHis-CNM Study Group; Laporte J, Cowling BS. Koch C, et al. Among authors: monseur a. Mol Ther Methods Clin Dev. 2020 May 4;17:1178-1189. doi: 10.1016/j.omtm.2020.04.022. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32514412 Free PMC article.