Results 21 to 30 of about 71,442 (257)
Health and Quality of Life Outcomes, 2022 Introduction Duchenne muscular dystrophy is a rare, progressive, life-limiting genetic neuromuscular condition that significantly impacts the quality of life of informal caregivers. Carer quality of life is measured using heterogeneous self-report scales,
Jill Carlton +2 more
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JAMA Neurology, 2020 This nonrandomized controlled trial analyzes safety, biological, and functional outcomes associated with the infusion of rAAVrh74.MHCK7.micro-dystrophin gene transfer in a small group of patients with Duchenne muscular dystrophy.
J. Mendell +19 more
semanticscholar +1 more source
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy
JAMA Network Open, 2022 This nonrandomized controlled trial examines efficacy of vamorolone treatment for Duchenne muscular dystrophy among boys compared with glucocorticoid treatment.
J. Mah +26 more
semanticscholar +1 more source
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Neurology, 2020 Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.
D. Frank +14 more
semanticscholar +1 more source
CRISPR Therapeutics for Duchenne Muscular Dystrophy
International Journal of Molecular Sciences, 2022 Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder with a prevalence of approximately 1 in 3500–5000 males. DMD manifests as childhood-onset muscle degeneration, followed by loss of ambulation, cardiomyopathy, and death in ...
Esra Erkut, T. Yokota
semanticscholar +1 more source
Molecular Therapy: Nucleic Acids, 2018 Duchenne muscular dystrophy (DMD) is caused by mutations in DMD, resulting in loss of dystrophin, which is essential to muscle health. DMD “exon skipping” uses anti-sense oligo-nucleotides (AONs) to force specific exon exclusion during mRNA processing to
Derek W. Wang +8 more
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Implicit learning deficit in children with Duchenne muscular dystrophy: Evidence for a cerebellar cognitive impairment? [PDF]
PLoS ONE, 2018 This study aimed at comparing implicit sequence learning in individuals affected by Duchenne Muscular Dystrophy without intellectual disability and age-matched typically developing children.
Stefano Vicari +16 more
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Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis
European Journal of Epidemiology, 2020 Several studies indicate that prognosis for survival in Duchenne muscular dystrophy (DMD) has improved in recent decades. However, published evidence is inconclusive and some estimates may be obsolete due to improvements in standards of care, in ...
E. Landfeldt +5 more
semanticscholar +1 more source
The Egyptian Heart Journal, 2021 Background With the continuous improvement of the respiratory care of Duchenne muscular dystrophy patients, cardiac manifestations (heart failure and arrhythmias) become the leading causes of morbidity and mortality.
Mahmoud Shehta +4 more
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Al-Azhar Assiut Medical Journal, 2019 Background Patients with Duchenne muscular dystrophy (DMD) and Becker’s muscular dystrophy (BMD) may have asymptomatic cardiac involvement for years before the development of dilated cardiomyopathy and even showed normal conventional echocardiographic ...
Shaimaa A Habib, Mohie El Din T Mohamed
doaj +1 more source