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Data from: Influence of full-length dystrophin on brain volumes in mouse models of Duchenne muscular dystrophy
- 负责人：
- DOI：
- doi:10.5061/dryad.7s2dq
- 摘要：
- Duchenne muscular dystrophy (DMD) affects besides muscle also the brain, resulting in memory and behavioral problems. The consequences
Data from: Cardiac dysfunction in Duchenne muscular dystrophy is less frequent in patients with mutations in the dystrophin Dp116 coding region tha
- 负责人：
- DOI：
- doi:10.5061/dryad.2m614
- 摘要：
- Background: Duchenne muscular dystrophy (DMD), the most common inherited muscular disease in childhood, is caused by dystrophin deficiency bec
Data from: Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
- 负责人：
- DOI：
- doi:10.5061/dryad.1rd4hc7
- 摘要：
- mmatory drug, in Duchenne muscular dystrophy. Methods: An open-label, multiple-ascending dose study of vamorolone was conducted in 48 boys with Duchenne muscular dystrophy (4 to <7 year
Data from: Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
- 负责人：
- DOI：
- doi:10.5061/dryad.g003051
- 摘要：
- with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Methods Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly
Data from: Discovery of metabolic biomarkers for Duchenne Muscular Dystrophy within a natural history study
- 负责人：
- DOI：
- doi:10.5061/dryad.qg6q2
- 摘要：
- Serum metabolite profiling in Duchenne muscular dystrophy (DMD) may enable discovery of valuable molecular markers for disease pro
Data from: The effects of experimental sleep apnea on cardiac and respiratory functions in 6 and 18 Month Old dystrophic mdx mice
- 负责人：
- DOI：
- doi:10.5061/dryad.k6n5k
- 摘要：
- Duchenne muscular dystrophy (DMD) is a fatal disease where over 90% of patients succumb to respiratory or cardiac failure. Sleep apne
Data from: Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial
- 负责人：
- DOI：
- doi:10.5061/dryad.7651036
- 摘要：
- with Duchenne muscular dystrophy (DMD). Methods: The Halt Cardiomyopathy Progression (HOPE)-Duchenne trial is a phase I/II, randomized, controlled, open-label trial (NCT02485938
Data from: Discovery of potential urine-accessible metabolite biomarkers associated with muscle disease and corticosteroid response in the mdx mouse
- 负责人：
- 关键词：
- DOI：
- doi:10.5061/dryad.v42h23k
- 摘要：
- Urine is increasingly being considered as a source of biomarker development in Duchenne Muscular Dystrophy (DMD), a severe, life-limiting disorder
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