标题
Therapeutic developments for Duchenne muscular dystrophy
作者
关键词
-
出版物
Nature Reviews Neurology
Volume -, Issue -, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2019-05-30
DOI
10.1038/s41582-019-0203-3
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial
- (2019) Michael Taylor et al. NEUROLOGY
- Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
- (2019) Christopher E. Nelson et al. NATURE MEDICINE
- Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy
- (2019) Panteleimon D. Mavroudis et al. JOURNAL OF CLINICAL PHARMACOLOGY
- Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients
- (2018) Christian Leborgne et al. CELLULAR IMMUNOLOGY
- Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study
- (2018) Craig M McDonald et al. LANCET
- Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan
- (2018) David J Birnkrant et al. LANCET NEUROLOGY
- Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
- (2018) David J Birnkrant et al. LANCET NEUROLOGY
- Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management
- (2018) David J Birnkrant et al. LANCET NEUROLOGY
- Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
- (2018) Silvana M.G. Jirka et al. MOLECULAR THERAPY
- Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
- (2018) Dongsheng Duan MOLECULAR THERAPY
- Deflazacort vs prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post hoc analysis from the ACT DMD trial
- (2018) Perry B Shieh et al. MUSCLE & NERVE
- Why dystrophin quantification is key in the eteplirsen saga
- (2018) Annemieke Aartsma-Rus et al. Nature Reviews Neurology
- Eteplirsen treatment for Duchenne muscular dystrophy
- (2018) Jay S. Charleston et al. NEUROLOGY
- A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy
- (2018) Nathalie Goemans et al. NEUROMUSCULAR DISORDERS
- Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017
- (2018) Volker Straub et al. NEUROMUSCULAR DISORDERS
- Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
- (2018) Hirofumi Komaki et al. Science Translational Medicine
- Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes
- (2018) Eric P. Hoffman et al. STEROIDS
- Therapeutic Mechanisms of Glucocorticoids
- (2018) Jolien Vandewalle et al. TRENDS IN ENDOCRINOLOGY AND METABOLISM
- Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy
- (2018) Craig M. McDonald et al. Annals of Clinical and Translational Neurology
- CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo
- (2018) Benjamin L. Duchêne et al. MOLECULAR THERAPY
- DMD CLINICAL THERAPIES I
- (2018) K. Wagner et al. NEUROMUSCULAR DISORDERS
- Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
- (2018) Laurie S. Conklin et al. PHARMACOLOGICAL RESEARCH
- Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
- (2018) Leonela Amoasii et al. SCIENCE
- Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment
- (2018) Michael J. Gait et al. Nucleic Acid Therapeutics
- A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe
- (2018) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- A Novel NF-κB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients With Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects
- (2017) Joanne M. Donovan et al. JOURNAL OF CLINICAL PHARMACOLOGY
- Reduced serum myostatin concentrations associated with genetic muscle disease progression
- (2017) Peter M. Burch et al. JOURNAL OF NEUROLOGY
- Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
- (2017) Craig M McDonald et al. LANCET
- Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5 ch and AAV1
- (2017) Anna Majowicz et al. MOLECULAR THERAPY
- Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides
- (2017) Naoki Iwamoto et al. NATURE BIOTECHNOLOGY
- A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy
- (2017) Ronald G. Victor et al. NEUROLOGY
- Stunning pharmacological properties of DS-5141b, an antisense oligonucleotide consisting of 2'-O,4'-C-ethylene-bridged nucleic acids and 2'-O-methyl RNA, on dystrophin mRNA exon skipping
- (2017) K. Takaishi et al. NEUROMUSCULAR DISORDERS
- A Phase I, single- and repeated-dose study of TAS-205, a novel inhibitor of hematopoietic prostaglandin D synthase, in patients with Duchenne muscular dystrophy
- (2017) H. Komaki et al. NEUROMUSCULAR DISORDERS
- Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy
- (2017) Thomas Meier et al. NEUROMUSCULAR DISORDERS
- Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: results of a two-year open-label extension trial
- (2017) Subha V. Raman et al. Orphanet Journal of Rare Diseases
- Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
- (2017) Yusuke Echigoya et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
- (2017) Virginie Mariot et al. Nature Communications
- Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
- (2017) Niclas E. Bengtsson et al. Nature Communications
- FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice
- (2017) Ngoc Lu-Nguyen et al. Molecular Therapy-Nucleic Acids
- 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
- (2017) Michael St. Andre et al. Skeletal Muscle
- The golden retriever model of Duchenne muscular dystrophy
- (2017) Joe N. Kornegay Skeletal Muscle
- Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
- (2016) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
- (2016) Courtney S. Young et al. Cell Stem Cell
- Intra‐arterial transplantation of HLA‐matched donor mesoangioblasts in Duchenne muscular dystrophy
- (2016) Giulio Cossu et al. EMBO Molecular Medicine
- The importance of genetic diagnosis for Duchenne muscular dystrophy
- (2016) Annemieke Aartsma-Rus et al. JOURNAL OF MEDICAL GENETICS
- Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
- (2016) Volker Straub et al. LANCET NEUROLOGY
- CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
- (2016) Li Xu et al. MOLECULAR THERAPY
- Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial
- (2016) Craig Campbell et al. MUSCLE & NERVE
- Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy
- (2016) Craig M. McDonald et al. NEUROMUSCULAR DISORDERS
- Histological effects of givinostat in boys with Duchenne muscular dystrophy
- (2016) Paolo Bettica et al. NEUROMUSCULAR DISORDERS
- Exon Snipping in Duchenne Muscular Dystrophy
- (2016) Dwi U. Kemaladewi et al. TRENDS IN MOLECULAR MEDICINE
- Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contractures
- (2016) Joe N. Kornegay et al. Skeletal Muscle
- Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study
- (2016) Patricia Hafner et al. PLoS One
- Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy
- (2016) Valeria Ricotti et al. PLoS One
- Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
- (2016) Ignazio Maggio et al. Scientific Reports
- A Review of Nebivolol Pharmacology and Clinical Evidence
- (2015) Justin Fongemie et al. DRUGS
- Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy
- (2015) G. Cossu et al. EMBO Molecular Medicine
- Cell therapy in muscular dystrophies: many promises in mice and dogs, few facts in patients
- (2015) Daniel Skuk et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial
- (2015) Gunnar M Buyse et al. LANCET
- A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
- (2015) Jerry R Mendell et al. MOLECULAR THERAPY
- European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene
- (2015) Manuel Haas et al. NEUROMUSCULAR DISORDERS
- Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy
- (2015) Tim Cheetham et al. NEUROPEDIATRICS
- A prudent path forward for genomic engineering and germline gene modification
- (2015) D. Baltimore et al. SCIENCE
- Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
- (2015) C. Long et al. SCIENCE
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells
- (2015) M. Tabebordbar et al. SCIENCE
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
- (2015) C. E. Nelson et al. SCIENCE
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy
- (2014) Doris G. Leung et al. ANNALS OF NEUROLOGY
- Effect of sildenafil on skeletal and cardiac muscle in Becker muscular dystrophy
- (2014) Nanna Witting et al. ANNALS OF NEUROLOGY
- Myostatin and the skeletal muscle atrophy and hypertrophy signaling pathways
- (2014) J. Rodriguez et al. CELLULAR AND MOLECULAR LIFE SCIENCES
- Therapeutic suppression of premature termination codons: Mechanisms and clinical considerations (Review)
- (2014) JOHN KARIJOLICH et al. INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE
- Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
- (2014) Thomas Voit et al. LANCET NEUROLOGY
- Ataluren treatment of patients with nonsense mutation dystrophinopathy
- (2014) Katharine Bushby et al. MUSCLE & NERVE
- PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy
- (2014) M. D. Nelson et al. NEUROLOGY
- Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function
- (2014) Rebecca L. Terry et al. PLoS One
- Adeno-associated viral vectors do not efficiently target muscle satellite cells
- (2014) Andrea LH Arnett et al. Molecular Therapy-Methods & Clinical Development
- The Anticancer Drug Tamoxifen Counteracts the Pathology in a Mouse Model of Duchenne Muscular Dystrophy
- (2013) Olivier M. Dorchies et al. AMERICAN JOURNAL OF PATHOLOGY
- Unknown
- (2013) Silvia Consalvi et al. MOLECULAR MEDICINE
- Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates
- (2013) Louis G. Chicoine et al. MOLECULAR THERAPY
- Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy
- (2013) Jerry R. Mendell et al. MUSCLE & NERVE
- Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice
- (2013) Silvana M.G. Jirka et al. Nucleic Acid Therapeutics
- Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency
- (2013) HaiFang Yin et al. Molecular Therapy-Nucleic Acids
- Vascular-targeted therapies for Duchenne muscular dystrophy
- (2013) James P Ennen et al. Skeletal Muscle
- Quantitative evaluation of the beneficial effects in the mdx mouse of epigallocatechin gallate, an antioxidant polyphenol from green tea
- (2012) Yoshiko Nakae et al. HISTOCHEMISTRY AND CELL BIOLOGY
- Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping
- (2012) Adeline Vulin et al. MOLECULAR THERAPY
- The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy
- (2012) A. M. Sbrocchi et al. OSTEOPOROSIS INTERNATIONAL
- Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophy
- (2012) Gunnar M. Buyse et al. PEDIATRIC PULMONOLOGY
- The Paradox of Muscle Hypertrophy in Muscular Dystrophy
- (2012) Joe N. Kornegay et al. Physical Medicine and Rehabilitation Clinics of North America
- Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy
- (2012) E. A. Martin et al. Science Translational Medicine
- Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
- (2012) Corinne Betts et al. Molecular Therapy-Nucleic Acids
- Targeting the Activin Type IIB Receptor to Improve Muscle Mass and Function in the mdx Mouse Model of Duchenne Muscular Dystrophy
- (2011) Emidio E. Pistilli et al. AMERICAN JOURNAL OF PATHOLOGY
- Follistatin Improves Skeletal Muscle Healing after Injury and Disease through an Interaction with Muscle Regeneration, Angiogenesis, and Fibrosis
- (2011) Jinhong Zhu et al. AMERICAN JOURNAL OF PATHOLOGY
- Membrane Sealant Poloxamer P188 Protects Against Isoproterenol Induced Cardiomyopathy in Dystrophin Deficient Mice
- (2011) Christopher F Spurney et al. BMC Cardiovascular Disorders
- Long-Term Systemic Myostatin Inhibition via Liver-Targeted Gene Transfer in Golden Retriever Muscular Dystrophy
- (2011) Lawrence T. Bish et al. HUMAN GENE THERAPY
- CINRG pilot trial of coenzyme Q10 in steroid-treated duchenne muscular dystrophy
- (2011) Christopher F. Spurney et al. MUSCLE & NERVE
- Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
- (2011) Nathalie M. Goemans et al. NEW ENGLAND JOURNAL OF MEDICINE
- Antibody-Directed Myostatin Inhibition Improves Diaphragm Pathology in Young but not Adult Dystrophic mdx Mice
- (2010) Kate T. Murphy et al. AMERICAN JOURNAL OF PATHOLOGY
- Carvedilol can Prevent Cardiac Events in Duchenne Muscular Dystrophy
- (2010) Tsuyoshi Matsumura et al. INTERNAL MEDICINE
- Dystrophin Immunity in Duchenne's Muscular Dystrophy
- (2010) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers†
- (2009) Natee Jearawiriyapaisarn et al. CARDIOVASCULAR RESEARCH
- Flavocoxid counteracts muscle necrosis and improves functional properties in mdx mice: A comparison study with methylprednisolone
- (2009) Sonia Messina et al. EXPERIMENTAL NEUROLOGY
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
- Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates
- (2009) J. Kota et al. Science Translational Medicine
- Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance
- (2008) G. M. Buyse et al. EUROPEAN HEART JOURNAL
Find the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
SearchAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started