Prevalence of Duchenne and Becker muscular dystrophies in the United States.
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What can Duchenne Connect teach us about treating Duchenne muscular dystrophy?The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence reviewAge at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STARnet.Using Administrative Data to Ascertain True Cases of Muscular Dystrophy: Rare Disease SurveillanceDuchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular DystrophySplice-switching antisense oligonucleotides as therapeutic drugsTesting the Feasibility of a Passive and Active Case Ascertainment System for Multiple Rare Conditions Simultaneously: The Experience in Three US StatesPerceived quality of life among caregivers of children with a childhood-onset dystrophinopathy: a double ABCX model of caregiver stressors and perceived resources.Application of the International Classification of Functioning, Disability and Health system to symptoms of the Duchenne and Becker muscular dystrophies.Pathoproteomic profiling of the skeletal muscle matrisome in dystrophinopathy associated myofibrosis.The prevalence of DICER1 pathogenic variation in population databases.Delayed onset of ambulation in boys with Duchenne muscular dystrophy: Potential use as an endpoint in clinical trials.Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy.Emerging new tools to study and treat muscle pathologies: genetics and molecular mechanisms underlying skeletal muscle development, regeneration, and disease.Secondary Conditions Among Males With Duchenne or Becker Muscular Dystrophy.Nanotherapy for Duchenne muscular dystrophy.Rehabilitative technology use among individuals with Duchenne/Becker muscular dystrophy.Factors Associated With Health-Related Quality of Life in Children With Duchenne Muscular Dystrophy.A rare subclinical or mild type of Becker muscular dystrophy caused by a single exon 48 deletion of the dystrophin gene.Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.Effective regeneration of dystrophic muscle using autologous iPSC-derived progenitors with CRISPR-Cas9 mediated precise correction.Duchenne and Becker Muscular Dystrophies: A Review of Animal Models, Clinical End Points, and Biomarker Quantification.Dystrophinopathy muscle biopsies in the genetic testing era: One center's data.A feasibility study using solution-focused coaching for health promotion in children and young people with Duchenne muscular dystrophy.Treating pediatric neuromuscular disorders: The future is now.Parental Reflections on the Diagnostic Process for Duchenne Muscular Dystrophy: A Qualitative Study.Duchenne and Becker muscular dystrophy in adolescents: current perspectives.Enabling the disabled: Call for intercepting disability surge in Pakistan.At the Crossroads of Clinical and Preclinical Research for Muscular Dystrophy-Are We Closer to Effective Treatment for Patients?Prevalence of muscular dystrophy in patients with muscular disorders in Tehran, Iran.A Novel Setup and Protocol to Measure the Range of Motion of the Wrist and the HandThe Importance of Biophysical and Biochemical Stimuli in Dynamic Skeletal Muscle Models
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P2860
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@ast
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@en
type
label
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@ast
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@en
prefLabel
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@ast
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@en
P2093
P2860
P50
P356
P1433
P1476
Prevalence of Duchenne and Becker muscular dystrophies in the United States.
@en
P2093
Charlotte M Druschel
Christopher Cunniff
Deborah J Fox
Dennis J Matthews
Emma Ciafaloni
F John Meaney
Gideon K D Zamba
Julie Bolen
Katherine A James
Kristin M Caspers Conway
P2860
P304
P356
10.1542/PEDS.2014-2044
P407
P577
2015-02-16T00:00:00Z