Results 71 to 80 of about 71,356 (167)
Nature Communications, 2017 Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9.
N. Bengtsson +8 more
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Outcome of Long-Term Corticosteroid Treatment in Duchenne Muscular Dystrophy
Pediatric Neurology Briefs, 2007 The clinical orthopedic effects of chronic daily corticosteroid treatment were evaluated by chart review in boys with genetically confirmed Duchenne muscular dystrophy (DMD) followed at the Ohio State University Muscular Dystrophy Clinic between 2000 and
J Gordon Millichap
doaj +1 more source
The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
Orphanet Journal of Rare Diseases, 2017 BackgroundDuchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal neuromuscular disorder, present from birth, which occurs almost exclusively in males. We have reviewed contemporary evidence of burden, epidemiology, illness costs and treatment
S. Ryder +7 more
semanticscholar +1 more source
Stem Cell Research, 2020 Duchenne muscular dystrophy (DMD) is a severe and rapidly progressive hereditary muscular disease with X-linked recessive inheritance, occurring mainly in males.
K.R. Valetdinova +8 more
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Pediatric Neurology Briefs, 1989 The clinical progression and effects of therapy in 283 boys with Duchenne dystrophy and ten with Becker dystrophy followed for up to ten years in a collaborative study are reported from the Departments of Neurology and Biostatistics, Washington ...
J Gordon Millichap
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Multiparametric Deep Learning Tissue Signatures for Muscular Dystrophy: Preliminary Results [PDF]
arXiv, 2019 A current clinical challenge is identifying limb girdle muscular dystrophy 2I(LGMD2I)tissue changes in the thighs, in particular, separating fat, fat-infiltrated muscle, and muscle tissue. Deep learning algorithms have the ability to learn different features by using the inherent tissue contrasts from multiparametric magnetic resonance imaging (mpMRI).
arxiv
Mechanical Design Improvement of a Passive Device to Assist Eating in People Living with Movement Disorders [PDF]
Rehabilitation Engineering Society of North America (now
Rehabilitation Engineering and Assistive Technology Society of North America
(RESNA), 2020, 2020 Many people living with neurological disorders, such as cerebral palsy, stroke, muscular dystrophy or dystonia experience upper limb impairments (muscle spasticity, loss of selective motor control, muscle weakness or tremors) and have difficulty to eat independently.
arxiv
Exon-skipping advances for Duchenne muscular dystrophy.
Human Molecular Genetics, 2018 Duchenne muscular dystrophy (DMD) is a fatal genetic disorder characterized by progressive muscle wasting that has currently no cure. Exon-skipping strategy represents one of the most promising therapeutic approaches that aim to restore expression of a ...
L. Echevarría +2 more
semanticscholar +1 more source
Distrofias musculares en el paciente adulto
Revista Médica Clínica Las Condes, 2018 RESUMEN: Las distrofias musculares son un grupo de trastornos hereditarios, degenerativos, progresivos del músculo estriado, cuya manifestación cardinal es la debilidad de la musculatura estriada esquelética.
Nicholas Earle, MD +1 more
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