… New advances in gene therapy in muscular dystrophies and motor neuron disorders. …
was the only option for management of muscular dystrophies and motor neuron disorders (MNDs). …

… by DMD gene mutations, which results in muscular degeneration … Muscular Dystrophy (BMD),
also caused by the DMD gene … Targeting epigenetic modifiers could be beneficial for …

… copy of SMN2 as occurs with the modifier variants described within the gene. In fact, several
… by additional SMA genetic modifiers. Considering other possible SMA modifier genes, aside …

… the skeletal muscle causes facioscapulohumeral muscular dystrophy (FSHD). … therapies.
The unusual evolutionary origin of DUX4, its extensive epigenetic and post-transcriptional gene …

… However, this pragmatic proposition does not incorporate the presence of possible genetic
modifiers in SMA other than the number of SMN2 copies that can mitigate or exacerbate the …

… translation of gene selective approaches possible, based on RNA interference or viral gene
reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and …

… treatment of various diseases. As a result, there has been an increasing interest in the use of
these therapies to … death, for instance, spinal muscular atrophy (SMA) or amyotrophic lateral …

… muscle wasting disease, Duchenne Muscular Dystrophy (DMD)… from frame-shift mutations
in the dystrophin gene [5]. The … skeletal muscle membrane causing the progression of muscle …

… Plastin 3 is a protective modifier of autosomal recessive spinal muscular atrophy. Science.
2008; 320(5875): 524–527, doi: 10.1126/science.1155085, indexed in Pubmed: 18440926. …

Purpose of review This review aims to provide a summary of the pathophysiology, clinical
presentation and management options for facioscapulohumeral dystrophy (FSHD). We discuss …