… We will provide an update on the mechanisms that can result in muscular dystrophy and
an indication on how experimental therapeutic approaches are rapidly advancing. …

… D4Z4 chromatin components and the facioscapulohumeral muscular dystrophy (FSHD) …
) to 1 unit, chromatin modifiers (eg, SMCHD1) have less additional effect on the repression of …

… Genetic modifiers of SMA have been … (a genetic modifier that has a negative effect on the
severity of SMA) in a combinatorial approach with suboptimal SMN-targeted ASO treatment …

… mRNA splicing modifier, was approved as first-ever therapy of spinal muscular atrophy (SMA…
diseases such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD)…

… strong evidence that genetic modifiers influence outcome. To identify genetic modifier loci,
we used … affected mouse strains with muscular dystrophy. Using the Sgcg model of limb girdle …

… Becker muscular dystrophy). In this review, we outline the current therapeutic approaches
… The more likely explanation is the existence of genetic and environmental modifiers that are …

… muscular dystrophy (DMD) are summarized. Emphasis is given to compounds acting
downstream of dystrophin, … are gene product modifiers that act on RNA to correct mutations in the …

… atrophy, which are both antisense oligonucleotides that modify pre-mRNA … new genetic
therapies and ongoing clinical trials for Duchenne muscular dystrophy, spinal muscular atrophy, …

… Latent TGFβ binding protein 4 (LTBP4) has been identified as a genetic modifier that
correlated with age of ambulation loss. In, particular, a haplotype constituted by four SNPs has …

… for the permanent correction of diverse genetic … of genetic mutations that cause muscular
dystrophies. Finally, we highlight current cell- and animal-based studies of muscular dystrophy …