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Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy.
- Published in:
- Journal of Personalized Medicine, 2020, v. 10, n. 4, p. 236, doi. 10.3390/jpm10040236
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- Publication type:
- Article
Antigen-driven T cell-macrophage interactions mediate the interface between innate and adaptive immunity in histidyl-tRNA synthetase-induced myositis.
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- Frontiers in Immunology, 2023, p. 1, doi. 10.3389/fimmu.2023.1238221
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- Publication type:
- Article
Exposure‐Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy.
- Published in:
- Journal of Clinical Pharmacology, 2020, v. 60, n. 10, p. 1385, doi. 10.1002/jcph.1632
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- Publication type:
- Article
Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy.
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- Journal of Clinical Pharmacology, 2019, v. 59, n. 7, p. 979, doi. 10.1002/jcph.1388
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- Publication type:
- Article
Gene therapy for muscular dystrophy reaches human clinical trial.
- Published in:
- Annals of Neurology, 2009, v. 66, n. 3, p. 267, doi. 10.1002/ana.21803
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- Publication type:
- Article
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.
- Published in:
- Annals of Neurology, 2005, v. 58, n. 1, p. 151
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- Publication type:
- Article
CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.
- Published in:
- Annals of Neurology, 2005, v. 58, n. 1, p. 151
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- Publication type:
- Article
Adult phosphorylase b kinase deficiency.
- Published in:
- Annals of Neurology, 1990, v. 28, n. 4, p. 529, doi. 10.1002/ana.410280410
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- Publication type:
- Article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 830, doi. 10.1038/mt.2011.59
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- Publication type:
- Article
915. High and Low Affinity Consensus Binding Sequences to Extracellular α7 Integrin X2B Subunit Detected Using Phage Display Biopanning.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S352, doi. 10.1016/j.ymthe.2006.08.1005
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- Publication type:
- Article
574. Models of Cancer Cachexia: Treatment with Inhibition of NF-kB.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S221, doi. 10.1016/j.ymthe.2006.08.647
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- Publication type:
- Article
1086. Patterns of Gene Expression from In Utero Delivery of AAV Serotype 1
- Published in:
- 2005
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- Publication type:
- Abstract
Sustained Muscle Expression of Dystrophin from a High-Capacity Adenoviral Vector with Systemic Gene Transfer of T Cell Costimulatory Blockade
- Published in:
- Molecular Therapy, 2004, v. 10, n. 4, p. 688, doi. 10.1016/j.ymthe.2004.07.020
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- Publication type:
- Article
Deubiquitinating enzyme A20 negatively regulates NF-κB signaling in skeletal muscle in mdx mice.
- Published in:
- FASEB Journal, 2012, v. 26, n. 2, p. 587, doi. 10.1096/fj.11-189829
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- Publication type:
- Article
Cellular caspase-8-1ike inhibitory protein (cFLIP) prevents inhibition of muscle cell differentiation induced by cancer cells.
- Published in:
- FASEB Journal, 2006, v. 20, n. 14, p. 2570, doi. 10.1096/fj.06-6347fje
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- Publication type:
- Article
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
- Published in:
- 2020
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- Publication type:
- journal article
Front Cover, Volume 40, Issue 11.
- Published in:
- Human Mutation, 2019, v. 40, n. 11, p. i, doi. 10.1002/humu.23934
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- Publication type:
- Article
GAA variants and phenotypes among 1,079 patients with Pompe disease: Data from the Pompe Registry.
- Published in:
- Human Mutation, 2019, v. 40, n. 11, p. 2146, doi. 10.1002/humu.23878
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- Publication type:
- Article
Muscle glycogenosis with low phosphorylase kinase activity: mutations in PHKA1, PHKG1 or six other candidate genes explain only a minority of cases.
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- European Journal of Human Genetics, 2003, v. 11, n. 7, p. 516, doi. 10.1038/sj.ejhg.5200996
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- Publication type:
- Article
Concerns Regarding Therapeutic Implications of Very Low‐Level Dystrophin.
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- Annals of Neurology, 2021, v. 90, n. 1, p. 176, doi. 10.1002/ana.26097
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- Publication type:
- Article
Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy.
- Published in:
- Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-48548-9
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- Publication type:
- Article
Findings from the Longitudinal CINRG Becker Natural History Study.
- Published in:
- Journal of Neuromuscular Diseases, 2024, v. 11, n. 1, p. 201, doi. 10.3233/JND-230178
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- Publication type:
- Article
Reply to F. Muntoni et al.: "In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy
- Published in:
- 2023
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- Publication type:
- Letter
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
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- Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 439, doi. 10.3233/JND-221656
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- Publication type:
- Article
Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy.
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- Journal of Neuromuscular Diseases, 2023, v. 10, n. 3, p. 349, doi. 10.3233/JND-221527
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- Publication type:
- Article
Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
- Published in:
- Journal of Neuromuscular Diseases, 2022, v. 9, n. 4, p. 493, doi. 10.3233/JND-220811
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- Publication type:
- Article
The CINRG Becker Natural History Study: Baseline characteristics.
- Published in:
- 2020
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- Publication type:
- journal article
Twenty-year follow-up of newborn screening for patients with muscular dystrophy.
- Published in:
- 2016
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- Publication type:
- journal article
Development of novel NEMO-binding domain mimetics for inhibiting IKK/NF-κB activation.
- Published in:
- PLoS Biology, 2018, v. 16, n. 6, p. 1, doi. 10.1371/journal.pbio.2004663
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- Publication type:
- Article
Systemic Delivery of a High-Capacity Adenoviral Vector Expressing Mouse CTLA4Ig Improves Skeletal Muscle Gene Therapy
- Published in:
- Molecular Therapy, 2002, v. 6, n. 3, p. 369, doi. 10.1006/mthe.2002.0676
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- Publication type:
- Article
Immune Response to Full-Length Dystrophin Delivered to Dmd Muscle by a High-Capacity Adenoviral Vector
- Published in:
- Molecular Therapy, 2002, v. 6, n. 3, p. 359, doi. 10.1006/mthe.2002.0675
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- Publication type:
- Article
REPORTS.
- Published in:
- Human Molecular Genetics, 1994, v. 3, n. 11, p. 1983
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- Publication type:
- Article
The companions: regulatory T cells and gene therapy.
- Published in:
- Immunology, 2009, v. 127, n. 1, p. 1, doi. 10.1111/j.1365-2567.2009.03069.x
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- Publication type:
- Article
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
- Published in:
- JAMA Network Open, 2022, v. 5, n. 1, p. e2144178, doi. 10.1001/jamanetworkopen.2021.44178
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- Publication type:
- Article
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
- Published in:
- Journal of Neurology, 2024, v. 271, n. 4, p. 1787, doi. 10.1007/s00415-023-12096-0
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- Publication type:
- Article
D-Amino Acid Substitution of Peptide-Mediated NF-κB Suppression in mdx Mice Preserves Therapeutic Benefit in Skeletal Muscle, but Causes Kidney Toxicity.
- Published in:
- Molecular Medicine, 2015, v. 21, n. 1, p. 442, doi. 10.2119/molmed.2013.00141
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- Publication type:
- Article
Adeno-associated Virus Serotype 8 (AAV8) Delivery of Recombinant A20 to Skeletal Muscle Reduces Pathological Activation of Nuclear Factor (NF)-κB in Muscle of mdx Mice.
- Published in:
- Molecular Medicine, 2012, v. 18, n. 12, p. 1527, doi. 10.2119/molmed.2012.00299
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- Publication type:
- Article
Effect of Nuclear Factor B Inhibition on Serotype 9 Adeno-Associated Viral (AAV9) Minidystrophin Gene Transfer to the mdx Mouse.
- Published in:
- Molecular Medicine, 2012, v. 18, n. 3, p. 466, doi. 10.2119/molmed.2011.00404
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- Publication type:
- Article
Rapamycin Ameliorates Dystrophic Phenotype in mdx Mouse Skeletal Muscle.
- Published in:
- Molecular Medicine, 2011, v. 17, n. 9/10, p. 917, doi. 10.2119/molmed.2010.00256
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- Publication type:
- Article
Peptide-Based Inhibition of NF- κRescues Diaphragm Muscle Contractile Dysfunction in a Murine Model of Duchenne Muscular Dystrophy.
- Published in:
- Molecular Medicine, 2011, v. 17, n. 5/6, p. 508, doi. 10.2119/molmed.2010.00263
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- Publication type:
- Article
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
- Published in:
- JAMA Neurology, 2023, v. 80, n. 6, p. 558, doi. 10.1001/jamaneurol.2023.0552
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- Publication type:
- Article
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
- Published in:
- JAMA Neurology, 2022, v. 79, n. 10, p. 1005, doi. 10.1001/jamaneurol.2022.2480
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- Publication type:
- Article
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial.
- Published in:
- JAMA Neurology, 2020, v. 77, n. 8, p. 982, doi. 10.1001/jamaneurol.2020.1264
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- Publication type:
- Article
NF-κB inhibition delays DNA damage- induced senescence and aging in mice.
- Published in:
- Journal of Clinical Investigation, 2012, v. 122, n. 7, p. 2601, doi. 10.1172/JCI45785
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- Publication type:
- Article
Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children.
- Published in:
- Scientific Reports, 2016, p. 31727, doi. 10.1038/srep31727
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- Publication type:
- Article
Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.
- Published in:
- Human Molecular Genetics, 2020, v. 29, n. 15, p. 2481, doi. 10.1093/hmg/ddaa132
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- Publication type:
- Article
Correction: Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study.
- Published in:
- 2016
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- Publication type:
- Correction Notice
Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study.
- Published in:
- PLoS ONE, 2016, v. 11, n. 4, p. 1, doi. 10.1371/journal.pone.0153461
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- Publication type:
- Article
Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy.
- Published in:
- Muscle & Nerve, 2014, v. 50, n. 2, p. 250, doi. 10.1002/mus.24163
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- Publication type:
- Article
Parental attitudes toward newborn screening for Duchenne/Becker muscular dystrophy and spinal muscular atrophy.
- Published in:
- Muscle & Nerve, 2014, v. 49, n. 6, p. 822, doi. 10.1002/mus.24100
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- Publication type:
- Article