Works matching DE "TREATMENT of Duchenne muscular dystrophy"
Results: 262
Perceptions of Healthcare Quality in Duchenne Muscular Dystrophy: A Patient Experience Exploratory Study.
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- Healthcare (2227-9032), 2025, v. 13, n. 4, p. 412, doi. 10.3390/healthcare13040412
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- Article
An overview of recent US-approved gene therapies for Duchenne muscular dystrophy and their respective clinical development programs.
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- Drugs & Therapy Perspectives, 2023, v. 39, n. 4, p. 156, doi. 10.1007/s40267-023-00986-2
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- Article
Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 2: diseases of motor neuron and skeletal muscle.
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- 2019
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- journal article
Duchenne muscular dystrophy: overview and future challenges.
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- Current Neurology / Aktualno?ci Neurologiczne, 2017, v. 17, n. 3, p. 144, doi. 10.15557/AN.2017.0015
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- Article
Pre-clinical evaluation of N-acetylcysteine reveals side effects in the mdx mouse model of Duchenne muscular dystrophy.
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- Journal of Physiology, 2017, v. 595, n. 23, p. 7093, doi. 10.1113/JP274229
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- Article
Oral quercetin administration transiently protects respiratory function in dystrophin-deficient mice.
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- Journal of Physiology, 2016, v. 594, n. 20, p. 6037, doi. 10.1113/JP272057
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- Article
Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis.
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- 2017
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- journal article
Utrophin influences mitochondrial pathology and oxidative stress in dystrophic muscle.
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- Skeletal Muscle, 2017, v. 7, p. 1, doi. 10.1186/s13395-017-0139-5
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- Article
A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy and its humanized equivalent, domagrozumab (PF-06252616), increases muscle volume in cynomolgus monkeys.
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- Skeletal Muscle, 2017, v. 7, p. 1, doi. 10.1186/s13395-017-0141-y
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- Article
Treatment with rGDF11 does not improve the dystrophic muscle pathology of mdx mice.
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- Skeletal Muscle, 2016, v. 6, p. 1, doi. 10.1186/s13395-016-0092-8
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- Article
CD49d is a disease progression biomarker and a potential target for immunotherapy in Duchenne muscular dystrophy.
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- Skeletal Muscle, 2015, v. 5, p. 1, doi. 10.1186/s13395-015-0066-2
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- Article
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells.
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- Skeletal Muscle, 2015, v. 5, p. 1, doi. 10.1186/s13395-015-0062-6
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- Article
Increased sphingosine-1-phosphate improves muscle regeneration in acutely injured mdx mice.
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- Skeletal Muscle, 2013, v. 3, n. 1, p. 1, doi. 10.1186/2044-5040-3-20
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- Article
Neuromuscular conditions for physicians - what you need to know.
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- Clinical Medicine, 2016, v. 16, n. 3, p. 259, doi. 10.7861/clinmedicine.16-3-259
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- Article
The era of genomic medicine.
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- Clinical Medicine, 2013, v. 13, n. 6, p. 594, doi. 10.7861/clinmedicine.13-6-594
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- Article
The Growing Role of Noninvasive Ventilation in Patients Requiring Prolonged Mechanical Ventilation.
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- Respiratory Care, 2012, v. 57, n. 6, p. 900, doi. 10.4187/respcare.01692
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- Article
Is Noninvasive Ventilation Possible Only in New Jersey?
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- 2011
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- Letter
The physiological response of protease inhibition in dystrophic muscle.
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- Acta Physiologica, 2013, v. 208, n. 3, p. 234, doi. 10.1111/apha.12114
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Fatigue in young people with Duchenne muscular dystrophy.
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- 2020
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- journal article
A new era in the management of Duchenne muscular dystrophy.
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- 2019
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- journal article
Development and content validation of the Muscular Dystrophy Child Health Index of Life with Disabilities questionnaire for children with Duchenne muscular dystrophy.
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- 2019
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- journal article
Variations in Duchenne muscular dystrophy course in a multi-ethnic UK population: potential influence of socio-economic factors.
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- 2017
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- journal article
Outcomes in Duchenne muscular dystrophy: nature, nurture, culture-or all three?
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- 2017
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- journal article
Focus on the upper limb in Duchenne muscular dystrophy.
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- 2017
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- journal article
Duchenne muscular dystrophy and the stomatognathic system.
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- 2016
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- journal article
Shedding light on the leukodystrophies.
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- 2016
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- journal article
Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice.
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- FASEB Journal, 2008, v. 22, n. 2, p. 477, doi. 10.1096/fj.07-8673com
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- Article
Are Corticosteroids Effective in Duchenne Muscular Dystrophy?
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- Indian Pediatrics, 2008, v. 45, n. 5, p. 401
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- Article
Rapamycin Ameliorates Dystrophic Phenotype in mdx Mouse Skeletal Muscle.
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- Molecular Medicine, 2011, v. 17, n. 9/10, p. 917, doi. 10.2119/molmed.2010.00256
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- Article
Stem-cell biology: A move in the right direction.
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- Nature, 2006, v. 444, n. 7119, p. 552, doi. 10.1038/nature05406
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- Article
Prostaglandin EP2 receptor downstream of Notch signaling inhibits differentiation of human skeletal muscle progenitors in differentiation conditions.
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- Communications Biology, 2020, v. 3, n. 1, p. 1, doi. 10.1038/s42003-020-0904-6
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- Article
Non-ambulatory Duchenne muscular dystrophy: observations, interventions, and outcomes of a single case.
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- Paediatrica Indonesiana, 2022, v. 62, n. 3, p. 208, doi. 10.14238/pi62.3.2022.208-16
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- Article
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
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- Human Genetics, 2016, v. 135, n. 9, p. 1029, doi. 10.1007/s00439-016-1725-z
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- Article
Skipping Multiple Exons to Treat DMD--Promises and Challenges.
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- Biomedicines, 2018, v. 6, n. 1, p. 1, doi. 10.3390/biomedicines6010001
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- Article
DMD Open‐access Variant Explorer (DOVE): A scalable, open‐access, web‐based tool to aid in clinical interpretation of genetic variants in the DMD gene.
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- Molecular Genetics & Genomic Medicine, 2019, v. 7, n. 1, p. N.PAG, doi. 10.1002/mgg3.510
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- Article
Author Correction: High-throughput identification of post-transcriptional utrophin up-regulators for Duchenne muscle dystrophy (DMD) therapy.
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- 2020
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- Correction Notice
Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy.
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- Journal of Personalized Medicine, 2018, v. 8, n. 4, p. 38, doi. 10.3390/jpm8040038
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- Article
Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges.
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- Journal of Personalized Medicine, 2018, v. 8, n. 4, p. 41, doi. 10.3390/jpm8040041
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- Article
Corticosteroid Treatment Impact on Spinal Deformity in Duchenne Muscular Dystrophy.
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- ISRN Otolaryngology, 2014, p. 1, doi. 10.1155/2014/965235
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- Article
Development of clinical practice guidelines for allied health and nursing assessment and management of Duchenne muscular dystrophy.
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- Disability & Rehabilitation, 2022, v. 44, n. 19, p. 5450, doi. 10.1080/09638288.2021.1936221
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- Article
The effects of calf massage in boys with Duchenne muscular dystrophy: a prospective interventional study.
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- Disability & Rehabilitation, 2021, v. 43, n. 26, p. 3803, doi. 10.1080/09638288.2020.1753829
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- Article
Evaluation of hand orthoses in Duchenne muscular dystrophy.
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- Disability & Rehabilitation, 2018, v. 40, n. 23, p. 2824, doi. 10.1080/09638288.2017.1347721
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- Article
Nanopatterned Human iPSC-Based Model of a Dystrophin-Null Cardiomyopathic Phenotype.
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- Cellular & Molecular Bioengineering, 2015, v. 8, n. 3, p. 320, doi. 10.1007/s12195-015-0413-8
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- Article
Lived Experience of Mothers with Duchenne Muscular Dystrophy Children, Kathmandu.
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- Journal of Institute of Medicine Nepal (JIOMN), 2015, v. 37, n. 2, p. 85, doi. 10.59779/jiomnepal.741
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- Article
What Percentage of Patients with Duchene Muscular Dystrophy are Potentially Treatable with Gene Therapies?
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- Annals of Indian Academy of Neurology, 2021, v. 24, n. 4, p. 601, doi. 10.4103/aian.aian_806_20
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- Article
Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases.
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- Annals of Indian Academy of Neurology, 2018, v. 21, n. 1, p. 3, doi. 10.4103/aian.AIAN_298_17
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- Article
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy: Quality of Evidence Concerns-an Alternative Viewpoint.
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- Pharmacotherapy, 2017, v. 37, n. 10, p. e109, doi. 10.1002/phar.1996
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- Article
Elemental analysis of biological tissues of Dmd/J and C57BL/6J mice strains investigated by neutron activation analysis.
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- Journal of Radioanalytical & Nuclear Chemistry, 2013, v. 296, n. 1, p. 579, doi. 10.1007/s10967-012-2005-6
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- Article
Make or break for first splice-modulating agents.
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- Nature Reviews Drug Discovery, 2013, v. 12, n. 11, p. 813, doi. 10.1038/nrd4151
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- Article
Market watch: Upcoming market catalysts in Q4 2013.
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- Nature Reviews Drug Discovery, 2013, v. 12, n. 10, p. 731, doi. 10.1038/nrd4136
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- Article