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REVISTA DE NEUROLOGIA, ISSN 0210-0010, 02/2018, Volume 66, Issue 4, pp. 135 - 135
Journal Article
JAMA Neurology, ISSN 2168-6149, 07/2018, Volume 75, Issue 7, pp. 791 - 793
Journal Article
PM and R, ISSN 1934-1482, 2019
Duchenne muscular dystrophy (DMD) is a neuromuscular condition characterized by progressive muscle weakness and contractures resulting in loss of ambulation in... 
tendoachilles lengthening | contracture | rehabilitation | eteplirsen | Duchenne muscular dystrophy
Journal Article
Science Translational Medicine, ISSN 1946-6234, 11/2017, Volume 9, Issue 418, p. eaan8081
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by mutations in the dystrophin gene. The majority of DMD mutations are... 
MEDICINE, RESEARCH & EXPERIMENTAL | THERAPY | GENE | EXONIC SPLICING ENHANCERS | ADENOASSOCIATED VIRUS | MUSCLE | MYOBLASTS | MICE | DUCHENNE | MUTATIONS | ETEPLIRSEN | CELL BIOLOGY | Index Medicus
Journal Article
Annual Review of Pharmacology and Toxicology, ISSN 0362-1642, 1/2019, Volume 59, Issue 1, pp. 605 - 630
Journal Article
Therapeutic Advances in Neurological Disorders, ISSN 1756-2856, 8/2017, Volume 10, Issue 8, pp. 293 - 296
Journal Article
Annual Review of Medicine, ISSN 0066-4219, 1/2019, Volume 70, Issue 1, pp. 307 - 321
Journal Article
Journal of Molecular and Cellular Cardiology, ISSN 0022-2828, 12/2018, Volume 125, pp. 29 - 38
Duchenne muscular dystrophy is a severe muscle wasting disease, characterized by a severely reduced lifespan in which cardiomyopathy is one of the leading... 
Heart | Low dystrophin levels | Exercise | Magnetic resonance imaging | Cardiomyopathy | FIBROSIS | CARDIAC & CARDIOVASCULAR SYSTEMS | MOUSE | INVOLVEMENT | MODEL | CARRIERS | MDX MICE | CHILDREN | CELL BIOLOGY | DIAPHRAGM | ETEPLIRSEN | Duchenne muscular dystrophy | Cardiac patients | Muscles | Running | Creatine | Dystrophin | Histochemistry
Journal Article
Orphan Drugs: Research and Reviews, ISSN 2230-6161, 08/2015, Volume 2015, Issue Issue 1, pp. 51 - 60
Corrado Angelini, Elisabetta Tasca Neuromuscular Laboratory, Fondazione San Camillo Hospital IRCCS, Venice, Italy Abstract: Therapeutic trials studying... 
eteplirsen | eplerenone | ataluren | DMD | steroids | drisapersen
Journal Article
Journal of Medicinal Chemistry, ISSN 0022-2623, 10/2018, Volume 61, Issue 20, pp. 9004 - 9029
With the rise of novel biology and high potential target identification technologies originating from advances in genomics, medicinal chemists are... 
PLURIPOTENT STEM-CELLS | INDUCED PROTEIN-DEGRADATION | RESIDUE INCORPORATION | CHEMISTRY, MEDICINAL | LIBRARY TECHNOLOGY | DRUG DISCOVERY | ALLOSTERIC MODULATION | TERTIARY STRUCTURE | PHAGE SELECTION | SMALL-MOLECULE INHIBITION | STRUCTURE-BASED DESIGN
Journal Article
Respiratory Physiology & Neurobiology, ISSN 1569-9048, 04/2019, Volume 262, pp. 1 - 11
Duchenne muscular dystrophy(DMD) shows motor and respiratory impairment. 19 DMD patients (DMDG) (nine ambulatory and 10 non-ambulatory) were evaluated through... 
Spirometry | Lung function | motor skills | Muscular dystrophy | Duchenne | Genetic disease | CAPACITY | PHYSIOLOGY | 6-MINUTE WALK TEST | PULMONARY-FUNCTION | CHILDREN | PEAK COUGH FLOW | PRESSURE | YOUNG | THERAPY | MUSCLE STRENGTH | RESPIRATORY SYSTEM | ETEPLIRSEN | Duchenne muscular dystrophy | Analysis | Heart beat | Index Medicus
Journal Article
Neuromuscular Disorders, ISSN 0960-8966, 05/2018, Volume 28, Issue 5, pp. 427 - 442
Dystrophin is a sub-sarcolemmal component of skeletal muscle fibres and is enriched at the postsynaptic membrane of the neuromuscular junction (NMJ). In the... 
Synaptic transmission | Dystrophin | Neuromuscular junction | Duchenne muscular dystrophy | Acetylcholine receptor | mdx mice | MOUSE | DISORDERS | CARRIERS | NEUROSCIENCES | CLINICAL NEUROLOGY | EXON | MIVACURIUM | MUSCLE-FIBERS | DUCHENNE MUSCULAR-DYSTROPHY | THERAPIES | EXPRESSION | ETEPLIRSEN | Medical research | Utrophin | Muscles | Medicine, Experimental | Acetylcholine | Neurophysiology
Journal Article
Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy, ISSN 0277-0008, 10/2017, Volume 37, Issue 10, pp. e112 - e117
Journal Article
Journal Article
Molecular Therapy, ISSN 1525-0016, 11/2017, Volume 25, Issue 11, pp. 2561 - 2572
Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused by mutations in the ( ) gene. Exon skipping is a therapeutic approach... 
mdx52 mice | Becker muscular dystrophy | Exondys 51 | eteplirsen | exon skipping | Duchenne muscular dystrophy | clinical trial candidate screening | BMD | antisense morpholino | drisapersen | machine learning | hDMD/Dmd-null mice | MEDICINE, RESEARCH & EXPERIMENTAL | DIAGNOSIS | DESIGN | EFFICACY | DMD GENE | RESTORATION | PHASE-2 | OLIGONUCLEOTIDES | THERAPY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | MICE | ETEPLIRSEN | Exons | Humans | Oligonucleotides, Antisense - metabolism | Male | Muscle, Skeletal - metabolism | Reading Frames | Recovery of Function | RNA Splicing | Female | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Disease Models, Animal | Gene Expression | Morpholinos - genetics | Mice, Transgenic | Muscular Dystrophy, Duchenne - pathology | Morpholinos - metabolism | Animals | Oligonucleotides, Antisense - genetics | Dystrophin - genetics | Mice | Muscular Dystrophy, Duchenne - metabolism | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | Mutation | Genetic Therapy - methods | Performance evaluation | Medical research | Statistical analysis | Splicing | Antisense oligonucleotides | Muscular dystrophy | Proteins | Musculoskeletal system | Protein folding | Efficiency | Exon skipping | Duchenne's muscular dystrophy | Protein expression | Muscle function | Dystrophy | Dystrophin | Evacuations & rescues | Index Medicus | hDMD | Dmd-null mice | Original
Journal Article