Characterization of patients with Becker muscular dystrophy by histology, magnetic resonance imaging, function, and strength assessments.

"CONFLICT OF INTEREST G.P.C. has served on scientific advisory boards of Avexis, PTC Therapeutics, Italfarmaco, Sarepta, Roche, and Biogen. E.H.N. has worked as an investigator on the Italfarmaco SpA clinical trial described in this article. He also reports ad hoc consultancies for BioMarin, Summit, WAVE, and Regenxbio, and worked as an investigator in clinical trials for BioMarin, GSK, Lilly, Santhera, WAVE, Roche, NS Pharma, Reveragen, and Sarepta, all outside the submitted work. All reimbursements were received by the LUMC. H.E.K. reports grants from Netherlands Organization for Health Research and Development, grant 91716490, research support from Philips Healthcare, trial support from ImagingDMD‐UF, and has served as a consultant for Esperare and PTC Therapeutics (no personal fees collected, all payments to the LUMC). KV reports grants from the National Institutes of Health (R01 AR056973) and research service support from Sarepta Therapeutics, Catabasis Pharmaceuticals, PTC Therapeutics, Eli Lilly, Summit Therapeutics, ML Bio/VCU directed to the University of Florida. N.M.v.d.V. has worked as a subinvestigator in clinical trials of givinostat, WAVE, Roche, and NS Pharma, all outside the submitted work. All reimbursements were received by the LUMC (no personal fees collected). S.C. and P.U.B. are employees of Italfarmaco SpA, the sponsor of the study. The remaining authors have no conflicts of interest to disclose."

"Funding information Italfarmaco; Regione Lombardia"

"Eligible patients were men aged 18 to 65 years, inclusive, with a diagnosis of BMD confirmed by genetic testing (based on patient records), who were able to walk between 200 and 450 meters on a 6MWT, and who, if receiving a systemic corticosteroid, angiotensin‐ converting enzyme inhibitor, and/or α‐ or β‐adrenergic receptor blocker, were expecting no significant change in dose or dosing regimen immediately before the start of study treatment. Among the reasons for exclusion were: use of any pharmacologic treatment in the 3 months before study entry, other than corticosteroids, or surgery that may have an effect on muscle strength or function; symptomatic cardiomyopathy or heart failure (New York Heart Association class III or IV) or left ventricular ejection fraction <50% at screening or with heart transplant; and contraindications to muscle biopsy or MRI. All patients provided written informed consent before any study‐related procedure. Full inclusion and exclusion criteria are listed in the Supporting Information online. This study was approved by an independent ethics committee at each institution, and was performed in accordance with the principles of the Declaration of Helsinki and the International Conference on Harmonization notes for guidance on Good Clinical Practice (ICH/CPMP/135/95). The study is registered at ClinicalTrials.gov (NCT03238235)."