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 …
by mutations in the dystrophin (DMD) gene. Most patients die from respiratory failure or …
Sleep disordered breathing in Duchenne muscular dystrophy
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 …
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
Purpose Despite poor sleep quality being recognised in Duchenne Muscular Dystrophy,
reports from milder forms of Muscular Dystrophy (MD), and accompanied associations with …
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 …
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 …
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 …
treatment of many human diseases including neurogenetic disorders. Most neurogenetic …
Resting energy expenditure in adults with Becker's muscular dystrophy
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 …
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 …
working group on corticosteroid therapy in children with Duchenne muscular dystrophy in …
Uncovering sleep in young males with Duchenne muscular dystrophy
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 …
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 …
Duchenne muscular dystrophy (DMD), and to explore the correlation between them, so as to …