Reyngoudt, Harmen http://orcid.org/0000-0002-0777-2711
Marty, Benjamin
Boisserie, Jean-Marc
Le Louër, Julien
Koumako, Cedi
Baudin, Pierre-Yves
Wong, Brenda
Stojkovic, Tanya
Béhin, Anthony
Gidaro, Teresa
Allenbach, Yves
Benveniste, Olivier
Servais, Laurent
Carlier, Pierre G.
Funding for this research was provided by:
BioMarin Pharmaceutical (NCT01753804)
Jain Foundation (NCT01676077)
Roche (NCT0239183)
FP7 (Bioimage-NMD)
Article History
Received: 27 May 2020
Revised: 29 October 2020
Accepted: 6 November 2020
First Online: 21 November 2020
Compliance with ethical standards
:
: The scientific guarantor of this publication is Harmen Reyngoudt, PhD.
: HR, BM, JMB, JLL, CK, PYB, TS, AB, TG, YA, OB and PGC report no disclosures relevant to the manuscript; BW worked as an investigator of clinical trials of BioMarin (including the PRO-DMD-01 NCT01753804 natural history study that included the DMD cohorts in this study) and received honoraria from serving on the advisory board of BioMarin; LS worked as an investigator for the NCT02780492 DMD natural history study and the NCT0239183 SMA natural history study co-funded by Roche and he performed consultancy for Roche, Avexis, Cytokinetics, Biogen, Sarepta, Biomarin, Pfizer, Biophytis.
: No complex statistical methods were necessary for this paper.
: Written informed consent was obtained from all subjects (patients) in this study.
: Institutional Review Board approval was obtained.
: Some study subjects or cohorts have been previously reported inGidaro T, Reyngoudt H, Le Louër J et al (2020) Quantitative nuclear magnetic resonance imaging detects subclinical changes over 1 year in skeletal muscle of GNE myopathy. J Neurol 267:228-238.Willis TA, Hollingsworth KG, Coombs A et al (2013) Quantitative muscle MRI as an assessment tool for monitoring disease progression in LGMD2I: A multicentre longitudinal study. PLoS One 8:e70993.Carlier PG, Azzabou N, de Sousa PL et al (2015) Skeletal muscle quantitative nuclear magnetic resonance imaging follow-up of adult Pompe patients. J Inherit Metab Dis 38:565–572.Naarding KJ, Reyngoudt H, van Zwet EW et al (2020) MRI vastus lateralis fat fraction predicts loss of ambulation in Duchenne muscular dystrophy. Neurology 94:e1386-1394.Chabanon A, Seferian AM, Daron A et al (2018) Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One 13:e0201004.
: • retrospective• observational• multicenter study