Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
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Title
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
Authors
Keywords
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Journal
Annals of Clinical and Translational Neurology
Volume 7, Issue 12, Pages 2393-2408
Publisher
Wiley
Online
2020-12-08
DOI
10.1002/acn3.51235
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Note: Only part of the references are listed.- Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
- (2020) Paula R. Clemens et al. JAMA Neurology
- DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype
- (2018) Richard T. Wang et al. HUMAN MUTATION
- Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy
- (2018) Chantal Beekman et al. PLoS One
- 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
- Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy
- (2017) Akinori Nakamura JOURNAL OF HUMAN GENETICS
- The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
- (2017) S. Ryder et al. Orphanet Journal of Rare Diseases
- 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
- Trends in steroid therapy for Duchenne muscular dystrophy in Japan
- (2016) Fumi Takeuchi et al. MUSCLE & NERVE
- DMDgenotypes and loss of ambulation in the CINRG Duchenne Natural History Study
- (2016) Luca Bello et al. NEUROLOGY
- Improving clinical trial design for Duchenne muscular dystrophy
- (2015) Luciano Merlini et al. BMC Neurology
- Advances in genetic therapeutic strategies for Duchenne muscular dystrophy
- (2015) Simon Guiraud et al. EXPERIMENTAL PHYSIOLOGY
- A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy
- (2014) Jean K. Mah et al. NEUROMUSCULAR DISORDERS
- Understanding the Process of Fibrosis in Duchenne Muscular Dystrophy
- (2014) Yacine Kharraz et al. Biomed Research International
- Eteplirsen for the treatment of Duchenne muscular dystrophy
- (2013) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Dystrophin levels and clinical severity in Becker muscular dystrophy patients
- (2013) J. C. van den Bergen et al. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- Quantification of dystrophin immunofluorescence in dystrophinopathy muscle specimens
- (2012) L. E. Taylor et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
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