Micro-dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials

Low dystrophin variability between muscles and stable expression over time in Becker muscular dystrophy using capillary Western immunoassay

(2021) Z. Koeks et al.   Scientific Reports

The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review

(2021) Shelagh M. Szabo et al.   Orphanet Journal of Rare Diseases

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

(2020) Jerry R. Mendell et al.   JAMA Neurology

Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy

(2020) Yvan Feraudy et al.   ANNALS OF NEUROLOGY

Development of Novel Micro-dystrophins with Enhanced Functionality

(2019) Julian N. Ramos et al.   MOLECULAR THERAPY

Women’s preferences for alternative financial incentive schemes for breastfeeding: A discrete choice experiment

(2018) Frauke Becker et al.   PLoS One

Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy

(2017) Nalinda B. Wasala et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

(2017) Chady H. Hakim et al.   Molecular Therapy-Methods & Clinical Development

Why are some patients with Duchenne muscular dystrophy dying young: An analysis of causes of death in North East England

(2016) H.J.A. Van Ruiten et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

DMDgenotypes and loss of ambulation in the CINRG Duchenne Natural History Study

(2016) Luca Bello et al.   NEUROLOGY

Low dystrophin levels in heart can delay heart failure in mdx mice

(2014) Maaike van Putten et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology

(2012) Maaike van Putten et al.   PLoS One

The Dystrophin-Glycoprotein Complex in the Prevention of Muscle Damage

(2011) Jessica D. Gumerson et al.   JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY

AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice

(2011) Brian Bostick et al.   MOLECULAR THERAPY

Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice

(2010) Dejia Li et al.   PLoS One

The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins

(2010) Glen B. Banks et al.   PLoS Genetics

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

(2009) Yi Lai et al.   JOURNAL OF CLINICAL INVESTIGATION

Preservation of Muscle Force in Mdx3cv Mice Correlates with Low-Level Expression of a Near Full-Length Dystrophin Protein

(2008) Dejia Li et al.   AMERICAN JOURNAL OF PATHOLOGY

Prevention of Dystrophin-Deficient Cardiomyopathy in Twenty-One-Month-Old Carrier Mice by Mosaic Dystrophin Expression or Complementary Dystrophin/Utrophin Expression

(2007) Brian Bostick et al.   CIRCULATION RESEARCH