Therapeutic approaches to muscular dystrophy

Duchenne Muscular Dystrophy: Therapeutic Approaches to Restore Dystrophin

Therapeutic advances in muscular dystrophy

The muscular dystrophies comprise a heterogeneous group of genetic disorders that produce progressive skeletal muscle weakness and wasting. There has been rapid growth and change in our understanding of these disorders in recent years, and advances in basic science are being translated into increasing numbers of clinical trials. This review will discuss therapeutic developments in 3 of the most...

Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD)

FacioScapuloHumeral muscular Dystrophy (FSHD) is one of the most prevalent hereditary myopathies and is generally characterized by progressive muscle atrophy affecting the face, scapular fixators; upper arms and distal lower legs. The FSHD locus maps to a macrosatellite D4Z4 repeat array on chromosome 4q35. Each D4Z4 unit contains a DUX4 gene; the most distal of which is flanked by a polyadenyl...

Therapeutic trials in muscular dystrophy. 1. Gold in murine dystrophy.

A trial of sodium aurothiomalate as an antiproteinase drug in the treatment of murine muscular dystrophy is reported. A blind controlled comparison of high (25 microgram/10 g body weight) and low dose gold (5 microgram/10 g body weight) with saline-injected control animals was made, all injections being given three times weekly. The body weights and functional ability of the mice were assessed ...

Nanolipodendrosome-loaded glatiramer acetate and myogenic differentiation 1 as augmentation therapeutic strategy approaches in muscular dystrophy

BACKGROUND [Corrected] Muscular dystrophies consist of a number of juvenile and adult forms of complex disorders which generally cause weakness or efficiency defects affecting skeletal muscles or, in some kinds, other types of tissues in all parts of the body are vastly affected. In previous studies, it was observed that along with muscular dystrophy, immune inflammation was caused by inflammat...