Develo** DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skip** therapies

O Sheikh, T Yokota - Expert opinion on investigational drugs, 2021 - Taylor & Francis
Introduction Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …

Sleep disordered breathing in Duchenne muscular dystrophy

A LoMauro, MG D'Angelo, A Aliverti - Current neurology and neuroscience …, 2017 - Springer
This review aims to explain the inevitable imbalance between respiratory load, drive, and
muscular force that occurs in the natural aging of Duchenne muscular dystrophy and that …

Objective and subjective measures of sleep in men with Muscular Dystrophy

CI Morse, G Onambele-Pearson, B Edwards… - PLoS …, 2022 - journals.plos.org
Purpose Despite poor sleep quality being recognised in Duchenne Muscular Dystrophy,
reports from milder forms of Muscular Dystrophy (MD), and accompanied associations with …

Obesity Management in Youth with Duchenne Muscular Dystrophy: A Review of Metformin and Alternative Pharmacotherapies

V Goldman, A Ryabets-Lienhard, L Howard… - Childhood …, 2024 - liebertpub.com
Background: Individuals with Duchenne muscular dystrophy (DMD) have increased risk of
obesity from prolonged glucocorticoid use and progressive muscle weakness. Over 50 …

Nutritional practices in pediatric patients with neuromuscular disorders

E Chou, R Lindeback, H Sampaio… - Nutrition Reviews, 2020 - academic.oup.com
Children with neuromuscular disorders (NMDs) may experience a spectrum of nutritional
issues with adverse health consequences. This review summarizes the current …

Sleep disorders in childhood neurogenetic disorders

LBM Dosier, BV Vaughn, Z Fan - Children, 2017 - mdpi.com
Genetic advances in the past three decades have transformed our understanding and
treatment of many human diseases including neurogenetic disorders. Most neurogenetic …

Resting energy expenditure in adults with Becker's muscular dystrophy

MF Jacques, P Orme, J Smith, CI Morse - PloS one, 2017 - journals.plos.org
Purpose The purpose of this study was: 1) To compare Resting energy expenditure (REE) in
adult males with Becker's Muscular Dystrophy (BeMD, n= 21, 39±12 years) and healthy …

Choice of compound, dosage, and management of side effects for long-term corticosteroid treatment in Duchenne muscular dystrophy: Guidelines from the …

SF Carbonnel, I Dabaj, C de Montferrand, P Rippert… - Archives de …, 2024 - Elsevier
Abstract The French Society of Pediatric Neurology and the FILNEMUS network created a
working group on corticosteroid therapy in children with Duchenne muscular dystrophy in …

Uncovering sleep in young males with Duchenne muscular dystrophy

AG Hartman, L Terhorst, N Little… - European Journal of …, 2020 - Elsevier
Objectives Sleep health in rare disease is often overlooked due to the complex nature of the
disease. For males with Duchenne muscular dystrophy, sleep assessment is typically …

Cross-Sectional Study of Nutritional Status and Dietary Nutrient Intake in Children with Duchenne Muscular Dystrophy (DMD) in China

HE Miao, CAI **ao-tang, P Wen-tao, S Jie… - JOURNAL OF …, 2022 - ykxb.scu.edu.cn
Objective To investigate the dietary nutrient intake and the nutritional status of children with
Duchenne muscular dystrophy (DMD), and to explore the correlation between them, so as to …