Works matching DE "TREATMENT of Duchenne muscular dystrophy"
Results: 259
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
Deflazacort.
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- Journal of Postgraduate Medicine, 2009, v. 55, n. 4, p. 296, doi. 10.4103/0022-3859.58942
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- Article
Cost-Effectiveness of Ventricular Assist Device Destination Therapy for Advanced Heart Failure in Duchenne Muscular Dystrophy.
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- Pediatric Cardiology, 2018, v. 39, n. 6, p. 1242, doi. 10.1007/s00246-018-1889-5
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- Article
Under-recognition of Low Blood Pressure Readings in Patients with Duchenne Muscular Dystrophy.
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- Pediatric Cardiology, 2015, v. 36, n. 7, p. 1489, doi. 10.1007/s00246-015-1191-8
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- Article
Myocardial Fibrosis and Left Ventricular Dysfunction in Duchenne Muscular Dystrophy Carriers Using Cardiac Magnetic Resonance Imaging.
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- Pediatric Cardiology, 2015, v. 36, n. 7, p. 1495, doi. 10.1007/s00246-015-1192-7
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- Article
Predictive Value of Myocardial Delayed Enhancement in Duchenne Muscular Dystrophy.
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- Pediatric Cardiology, 2014, v. 35, n. 7, p. 1279, doi. 10.1007/s00246-014-0929-z
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- Article
Gene Silencing Method Based on DNA.
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- Journal of Cell & Molecular Research, 2023, v. 15, n. 1, p. 7, doi. 10.22067/jcmr.2023.84052.1077
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- Article
Marathon Pharmaceutical and Deflazacort: Charity or Chicanery?
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- Penn Bioethics Journal, 2016, v. 13, n. 1, p. 8
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- Article
Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy.
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- Child: Care, Health & Development, 2013, v. 39, n. 3, p. 449, doi. 10.1111/j.1365-2214.2012.01387.x
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- Article
Effect of Citrus aurantium L. Essential Oil on Muscle Regeneration in Mdx Mice.
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- International Journal of Morphology, 2011, v. 29, n. 4, p. 1357, doi. 10.4067/S0717-95022011000400048
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- Article
Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy.
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- 2007
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- journal article
Inhibiting miR‐25 through Tough Decoy Gene Therapy Improves Calcium Handling and Abrogates Cardiac Dysfunction in Aged MDX/UTRN KO Mice.
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- 2018
- Publication type:
- Abstract
A phase I study of TAS‐205 in patients with Duchenne muscular dystrophy.
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- Annals of Clinical & Translational Neurology, 2018, v. 5, n. 11, p. 1338, doi. 10.1002/acn3.651
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- Article
Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.
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- Annals of Clinical & Translational Neurology, 2018, v. 5, n. 8, p. 913, doi. 10.1002/acn3.579
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- Article
Pathophysiology and Therapy of Cardiac Dysfunction in Duchenne Muscular Dystrophy.
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- American Journal of Cardiovascular Drugs, 2011, v. 11, n. 5, p. 287, doi. 10.2165/11594070-000000000-00000
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- Article
Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy.
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- Pediatric Transplantation, 2010, v. 14, n. 7, p. 813, doi. 10.1111/j.1399-3046.2010.01377.x
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- Article
Epigenetic Reprogramming of Muscle Progenitors: Inspiration for Clinical Therapies.
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- Thrombosis, 2015, p. 1, doi. 10.1155/2016/6093601
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- Article
Comparison of Deflazacort and Prednisone in Duchenne Muscular Dystrophy.
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- Iranian Journal of Child Neurology, 2012, v. 6, n. 1, p. 5
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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
Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy—analysis of registry data.
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- European Heart Journal, 2021, v. 42, n. 20, p. 1976, doi. 10.1093/eurheartj/ehab054
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- Article
A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy.
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- 2019
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- journal article
L-CARNITINE SUPPLEMENTATION IN DUCHENNE MUSCULAR DYSTROPHY STEROIDNAÏVE PATIENTS: A PILOT STUDY.
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- Current Topics in Nutraceutical Research, 2013, v. 11, n. 3, p. 97
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- Article
Naproxcinod shows significant advantages over naproxen in the mdx model of Duchenne Muscular Dystrophy.
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- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-015-0311-0
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- Article
A comprehensive database of Duchenne and Becker muscular dystrophy patients (0-18 years old) in East China.
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- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-014-0220-7
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- Article
Effect of yoga as an add-on therapy in the modulation of heart rate variability in children with duchenne muscular dystrophy.
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- International Journal of Yoga, 2019, v. 12, n. 1, p. 55, doi. 10.4103/ijoy.IJOY_12_18
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- Article
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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- 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
Effect of Yoga and Ayurveda on Duchenne Muscular Dystrophy.
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- 2011
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- Publication type:
- Letter
Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy.
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- Journal of Neurology, 2009, v. 256, n. 5, p. 768, doi. 10.1007/s00415-009-5012-y
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- Article
Dystrophin expression in the mdx mouse restored by stem cell transplantation.
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- Nature, 1999, v. 401, n. 6751, p. 390, doi. 10.1038/43922
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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
Therapeutic drug monitoring of vancomycin in a patient with Duchenne muscular dystrophy (DMD): A case report.
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- 2016
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- Case Study
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
Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects.
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- Clinical Pharmacology in Drug Development, 2019, v. 8, n. 2, p. 172, doi. 10.1002/cpdd.645
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- Article
Transplacental injection of somite-derived cells in mdx mouse embryos for the correction of dystrophin deficiency.
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- Human Molecular Genetics, 2000, v. 9, n. 12, p. 1843, doi. 10.1093/hmg/9.12.1843
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- Article
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.
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- Pharmaceuticals (14248247), 2013, v. 6, n. 7, p. 813, doi. 10.3390/ph6070813
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- Article
The effects of knee-ankle-foot orthoses in the treatment of Duchenne muscular dystrophy: review of the literature.
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- Clinical Rehabilitation, 2000, v. 14, n. 4, p. 343, doi. 10.1191/0269215500cr319oa
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- Article
Electrical impedance myography for assessment of Duchenne muscular dystrophy.
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- 2017
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- journal article
Quantitative muscle ultrasound detects disease progression in Duchenne muscular dystrophy.
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- 2017
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- Publication type:
- journal article
Deflazacort for the treatment of Duchenne Dystrophy: A systematic review.
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- BMC Neurology, 2003, v. 3, p. 7, doi. 10.1186/1471-2377-3-7
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- Article
P2RX7 purinoceptor: a therapeutic target for ameliorating the symptoms of duchenne muscular dystrophy.
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- 2015
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- journal article
THE FDA'S DR. NOs.
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- Regulation, 2016, v. 39, n. 4, p. 22
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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
Cardiac resynchronization therapy in a young patient with Duchenne muscular dystrophy.
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- International Medical Case Reports Journal, 2015, v. 8, p. 173, doi. 10.2147/IMCRJ.S87512
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- Article
FDA Approval of Eteplirsen for Muscular Dystrophy.
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- 2017
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- Publication type:
- Letter
Novel Drugs Passed Muster in 2016.
- Published in:
- 2017
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- Publication type:
- journal article
Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy.
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- 2016
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- Publication type:
- journal article
News From the Food and Drug Administration.
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- JAMA: Journal of the American Medical Association, 2016, v. 316, n. 17, p. 1756, doi. 10.1001/jama.2016.15537
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- Article