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Neuromuscular Disorders, ISSN 0960-8966, 01/2018, Volume 28, Issue 1, pp. 4 - 15
Journal Article
MOLECULAR THERAPY, ISSN 1525-0016, 01/2018, Volume 26, Issue 1, pp. 132 - 147
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder caused by reading frame disrupting mutations in the DMD gene. Exon skipping... 
MEDICINE, RESEARCH & EXPERIMENTAL | SAFETY | PHAGE DISPLAY | RESTORATION | DEFICIENT MDX MICE | MUSCLE-CELLS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | 2'-O-METHYL PHOSPHOROTHIOATE | SKELETAL | PROTEINS | EXPRESSION | DRISAPERSEN
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
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
Brain & Development, ISSN 0387-7604, 2016, Volume 38, Issue 8, pp. 738 - 745
Journal Article
Expert Opinion on Orphan Drugs, ISSN 2167-8707, 09/2017, Volume 5, Issue 9, pp. 683 - 690
Introduction: DMD is most common form of severe childhood muscular dystrophy. The large size of the DMD gene challenges DNA repair or replacement. However,... 
drisapersen | dystrophin | eteplirsen | exon skipping | Duchenne muscular dystrophy | CELLS | SAFETY | MOUSE | ANTISENSE OLIGONUCLEOTIDE | RESTORATION | PHASE-2 | GENE | PRO051 | PHARMACOLOGY & PHARMACY | EXPRESSION
Journal Article
Paediatrics and Child Health, ISSN 1751-7222, 2017, Volume 27, Issue 6, pp. 271 - 275
Abstract There have been several significant recent advances in the diagnosis and management of children with neuromuscular disorders. These have been... 
Pediatrics | myotonic dystrophy | Charcot–Marie–Tooth | spinal muscular atrophy | children | muscle disease | Duchenne muscular dystrophy | Neuromuscular diseases | Care and treatment | Research | Children | Health aspects
Journal Article
Drugs of Today, ISSN 1699-3993, 03/2016, Volume 52, Issue 3, pp. 199 - 202
Highly anticipated as new disease-modifying treatments for Duchenne muscular dystrophy (DMD), therapeutics by BioMarin Pharmaceutical (Kyndrisa™; drisapersen)... 
Drisapersen | Duchenne muscular dystrophy | Eteplirsen | Oligonucleotides - therapeutic use | Muscular Dystrophy, Duchenne - drug therapy | United States | Humans | Drug Approval | Morpholinos | United States Food and Drug Administration
Journal Article
DRUGS OF THE FUTURE, ISSN 0377-8282, 09/2013, Volume 38, Issue 9, pp. 619 - 623
Duchenne muscular dystrophy (DMD) is a progressive disorder with no standard curative treatment. Current therapy focuses on steroids to slow muscle wastage and... 
MDX MOUSE | PRO-051 | Drisapersen sodium | Antisense oligoribonucleotides | SEQUENCES | Duchenne muscular dystrophy | ANTISENSE | PHARMACOLOGY & PHARMACY | RESTORATION | Dystrophin | MOLECULAR-BASIS
Journal Article
Developmental Medicine & Child Neurology, ISSN 0012-1622, 03/2019, Volume 61, Issue 3, pp. 292 - 297
Updated standards of care covering most clinical aspects of Duchenne muscular dystrophy (DMD) are available. Adequately controlled clinical trials have allowed... 
RESPIRATORY-FUNCTION | DIAGNOSIS | EFFICACY | GENE | DOUBLE-BLIND | PEDIATRICS | DMD | HEALTH | ATALUREN | DRISAPERSEN | CLINICAL NEUROLOGY
Journal Article
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