Height, weight, and body mass index trajectories and their correlation with functional outcome assessments in boys with Duchenne muscular dystrophy.

The analysis included 194 boys, but 196 were recruited and 2 were screened without dosing. Observations after a glucocorticoid dose reduction of 25% or more were excluded, affecting 128 observations from 50 boys. The 5-year correlation analysis had only 32 boys, and the 6-minute walk test was not analysed because of low numbers. The correlations were observational and do not show that height, weight, or BMI changes caused motor decline. Factors such as parental height and weight, nutrition monitoring, growth hormone levels, pregnancy history, and genetic modifiers were not analysed. Glucocorticoid regimen or type could not be switched during the trial, so real-world changes were not assessed. Participants had to be able to rise independently at screening, so findings may not apply to boys who had already lost that ability. Only prepubertal boys with DMD were included, so findings may not apply to girls or older pubertal boys.

UK Duchenne through Newcastle University funded MS. MG is part of the Medical Research Council UK and TREAT-NMD, participates in advisory boards for Pfizer, NS Pharma, Roche, Italfarmaco, and Santhera, collaborates with Edgewise and Sarepta, is or has been principal investigator for trials with Roche, Italfarmaco, Edgewise, Genethon, Dyne, Santhera, ReveraGen, Summit, Pfizer, and PTC Therapeutics, and has speaker honoraria from Italfarmaco, Dyne, Roche, and Novartis. LMW declares consultancy for Amgen, Ultragenyx, Kyowa Kirin, Angitia, Riche, Santhera, Catalyst, Biomarin, and Ipsen, and trial participation with Alexion, QED, Ultragenyx, Edgewise, ReveraGen, Ascendis, Roche, and Catalyst; these fund her institution. RCG reports grants from NIH, MDA, and Patient Project for Muscular Dystrophy Support, and others from PTC Therapeutics and Sarepta Therapeutics during the study. CLW has acted as a paid consultant to Roche and PTC Therapeutics. UJD reports grants from NIH National Institute of Arthritis and Musculoskeletal and Skin Diseases and NINDS, the US Department of Defence, the Natural Sciences and Engineering Research Council of Canada, and the Foundation to Eradicate Duchenne, and declares consultancy for ReveraGen Biopharma, Lupin Neurosciences, and iuvo Bioscience, with trial participation involving ReveraGen Biopharma. AM participated in SAB meetings for Summit, PTC Therapeutics, and Biogen and performs consultancy work, including training physiotherapists for a DMD trial, for Roche, Pfizer, PTC Therapeutics, Summit, Sarepta Therapeutics, Santhera, Italfarmaco, Amicus, Biogen, and Avexis. SCW declares consultancy for Novartis, Santhera, and Roche and speaker honoraria from Sandoz, Roche, Novo Nordisk, and Nutricia. The other authors declare no disclosures related to this project.

Do not read the 5-year correlations as proof that weight gain caused motor decline. Only 32 boys were analysed, the 6-minute walk test was not analysed at 5 years, and the study reports correlations, not causation.