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Journal Article
BioNanoScience, ISSN 2191-1630, 06/2017, Volume 7, Issue 2, pp. 386 - 389
Due to lack of effective therapies, muscular dystrophies became a focus for gene therapy. Multiple pre-clinical studies have shown successful restoration of... 
RNA editing | Ribonucleic acids | Restoration | Editing | In vitro testing | Dystrophy | Gene therapy | Muscular dystrophy
Journal Article
Journal Article
Fortschritte der Neurologie-Psychiatrie, 09/2018, Volume 86, Issue 9, p. 535
Muscular dystrophies (MD) are a clinically and genetically heterogeneous group of skeletal muscle-wasting diseases with progressive muscle weakness and... 
Muscular Dystrophies - therapy | Humans | Muscular Dystrophies - genetics | Genetic Therapy - methods
Journal Article
Muscle & Nerve, ISSN 0148-639X, 07/2013, Volume 48, Issue 1, pp. 55 - 67
Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history... 
phenotype | quality of life/psychology | child/preschool | locomotion | muscular dystrophies/Duchenne/physiopathology | muscle strength/physiology | health status | muscular dystrophies/therapy | respiratory function tests | adolescent | follow‐up studies | adult | humans | male | muscular dystrophies/classification | Humans | Male | Follow-up studies | Child/preschool | Locomotion | Phenotype | Quality of life/psychology | Health status | Muscular dystrophies/Duchenne/physiopathology | Muscular dystrophies/therapy | Muscular dystrophies/classification | Adolescent | Adult | Muscle strength/physiology | Respiratory function tests | PROFILES | DESIGN | 6-MINUTE WALK TEST | RELIABILITY | CLINICAL NEUROLOGY | psychology | Duchenne | muscular dystrophies | PREDNISONE THERAPY | GAIT | therapy | physiology | MUSCULAR-DYSTROPHY | STRENGTH | classification | NEUROSCIENCES | muscle strength | follow-up studies | physiopathology | BOYS | preschool | STAR AMBULATORY ASSESSMENT | quality of life | child | Outcome Assessment (Health Care) - methods | Glucocorticoids - therapeutic use | Prospective Studies | Child, Preschool | Diagnostic Techniques, Neurological - standards | Muscular Dystrophy, Duchenne - epidemiology | Young Adult | Biomedical Research - methods | Clinical Trials as Topic - standards | Child | Cross-Sectional Studies | Muscular Dystrophy, Duchenne - drug therapy | Treatment Outcome | International Cooperation | Disease Progression | Outcome Assessment (Health Care) - standards | Clinical Trials as Topic - methods | Longitudinal Studies | Biomedical Research - standards | Cohort Studies | Muscle Strength - physiology | Muscular Dystrophy, Duchenne - diagnosis | Clinical trials | Medicine, Experimental | Medical research | Corticosteroids | Steroids | Index Medicus | Neurologi | Pediatrics | strength | Pediatrik | prednisone therapy | reliability | profiles | boys | 6-minute walk test | Neurology | protocol | children | muscular-dystrophy | star ambulatory assessment
Journal Article
Journal of Reproduction and Contraception, ISSN 1001-7844, 03/2015, Volume 26, Issue 1, pp. 53 - 60
Progress of cell therapy for muscular dystrophies is slow. Although there are clinical trials of myoblast delivery, the result is not satisfactory. However,... 
cell differentiation | induced pluripotent stem cells (iPSCs) | skeletal muscle | muscular dystrophies | embryonic stem cells (ESCs) | Muscular dystrophies | Cell differentiation | Skeletal muscle | Embryonic stem cells (ESCs) | Induced pluripotent stem cells (iPSCs) | Embryonic stem cells | Transplantation | Stem cells
Journal Article
Nature Medicine, ISSN 1078-8956, 07/2004, Volume 10, Issue 7, pp. 696 - 703
Journal Article
Cell Death and Disease, ISSN 2041-4889, 11/2012, Volume 3, Issue 11, pp. e418 - e418
A resolutive therapy for Duchene muscular dystrophy, a severe degenerative disease of the skeletal muscle, is still lacking. Because autophagy has been shown... 
Therapy | Autophagy | Duchenne muscular dystrophy | MDX MOUSE | autophagy | therapy | FOXO3 | CELL BIOLOGY | SKELETAL-MUSCLE | PATHWAY | DOWNSTREAM | DEACETYLASE INHIBITORS | IN-VIVO | NITRIC-OXIDE | MICE | DIFFERENTIATION | Index Medicus
Journal Article