… muscle degeneration in general. This review will discuss different genetic forms of muscular
dystrophy with an emphasis on phenotypic variability and the presence of genetic modifiers …

… genetically based therapies targeting the primary defect of Duchenne muscular dystrophy. …
This review provides an update on the current genetically based therapies and clinical trials …

… reviews genetic modifier studies in DMD to date and discusses the effect of genetic modifiers
on … (inclusion of genotype-stratified subgroup analyses) and therapeutic approaches. The …

… muscular dystrophy (DMD) phenotype. This meta-analysis aims to estimate the association
of genetic … ) and secreted phosphoprotein 1 (SPP1) genes, among others, with age of loss of …

… An example of such a genetic modifier identified from a genome-wide screen is the … new
therapeutic targets and provide novel insights into disease pathogenesis, even though the effect …

… a large variety of factors is spinal muscular atrophy (SMA), selected herein to exemplify the
role of modifiers and the use of this knowledge gained in developing target-driven therapies. …

… Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive …
on the recent gene therapies for DMD that aim to compensate for dystrophin deficiency and the …

… genetic modifiers in DMD suggests that the ‘effect size’ of the … Effect size of genetic modifiers
can be quantitated in DMD by … Genetic modifiers to date all show about 1-2 years change in …

… We report a putative protective effect of DMD genetic modifiers on the development of
cardiac complications, that might aid in risk stratification if confirmed in independent cohorts. …

… (mdx) mice, have yielded valuable genetic modifiers of disease pathology.32 This Review
… consequences of genetic modifiers in the 2 most prevalent forms of muscular dystrophy in …