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。Latent process model of the 6-minute walk test in Duchenne muscular dystrophy A Bayesian approach to quantifying rare disease progression
Janelle L. Lennie et al.
JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS (2020)
Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy
Panteleimon D. Mavroudis et al.
JOURNAL OF CLINICAL PHARMACOLOGY (2019)
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy
Daniela J. Conrado et al.
JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS (2019)
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
Eric P. Hoffman et al.
NEUROLOGY (2019)
Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study
Craig M. McDonald et al.
LANCET (2018)
Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history
Harneet Arora et al.
MUSCLE & NERVE (2018)
Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes
Eric P. Hoffman et al.
STEROIDS (2018)
Cryptic glucocorticoid receptor-binding sites pervade genomic NF-κB response elements
William H. Hudson et al.
NATURE COMMUNICATIONS (2018)
Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit
Sen Chandra Sreetama et al.
MOLECULAR THERAPY (2018)
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
Laurie S. Conklin et al.
PHARMACOLOGICAL RESEARCH (2018)
Serum biomarkers of glucocorticoid response and safety in anti-neutrophil cytoplasmic antibody-associated vasculitis and juvenile dermatomyositis
Laurie S. Conklin et al.
STEROIDS (2018)
VBP15, a novel dissociative steroid compound, reduces NFκB-induced expression of inflammatory cytokines in vitro and symptoms of murine trinitrobenzene sulfonic acid-induced colitis
Jesse M. Damsker et al.
INFLAMMATION RESEARCH (2016)
Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy Report of the Guideline Development Subcommittee of the American Academy of Neurology
David Gloss et al.
NEUROLOGY (2016)
A novel dissociative steroid VBP15 reduces MUC5AC gene expression in airway epithelial cells but lacks the GRE mediated transcriptional properties of dexamethasone
Lindsay M. Garvin et al.
PULMONARY PHARMACOLOGY & THERAPEUTICS (2016)
Identification of Pathway-Specific Serum Biomarkers of Response to Glucocorticoid and Infliximab Treatment in Children with Inflammatory Bowel Disease
Christopher R. Heier et al.
CLINICAL AND TRANSLATIONAL GASTROENTEROLOGY (2016)
Duchenne muscular dystrophy
Eppie M. Yiu et al.
JOURNAL OF PAEDIATRICS AND CHILD HEALTH (2015)
Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study
Luca Bello et al.
NEUROLOGY (2015)
Immune-mediated pathology in Duchenne muscular dystrophy
Amy S. Rosenberg et al.
SCIENCE TRANSLATIONAL MEDICINE (2015)
VBP15, a Novel Anti-Inflammatory, is Effective at Reducing the Severity of Murine Experimental Autoimmune Encephalomyelitis
Blythe C. Dillingham et al.
CELLULAR AND MOLECULAR NEUROBIOLOGY (2015)
Establishing Good Practices for Exposure-Response Analysis of Clinical Endpoints in Drug Development
R. V. Overgaard et al.
CPT-PHARMACOMETRICS & SYSTEMS PHARMACOLOGY (2015)
VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects
Christopher R. Heier et al.
EMBO MOLECULAR MEDICINE (2013)
THE 6-MINUTE WALK TEST AND OTHER CLINICAL ENDPOINTS IN DUCHENNE MUSCULAR DYSTROPHY: RELIABILITY, CONCURRENT VALIDITY, AND MINIMAL CLINICALLY IMPORTANT DIFFERENCES FROM A MULTICENTER STUDY
Craig M. McDonald et al.
MUSCLE & NERVE (2013)
VBP15, a Glucocorticoid Analogue, Is Effective at Reducing Allergic Lung Inflammation in Mice
Jesse M. Damsker et al.
PLOS ONE (2013)
THE 6-MINUTE WALK TEST IN DUCHENNE/BECKER MUSCULAR DYSTROPHY: LONGITUDINAL OBSERVATIONS
Craig M. McDonald et al.
MUSCLE & NERVE (2010)
Early onset of inflammation and later involvement of TGFβ in Duchenne muscular dystrophy
YW Chen et al.
NEUROLOGY (2005)