Early pathological signs in young dysf −/− mice are improved by halofuginone

Muscular dystrophies (MDs) are genetically inherited myogenic disorders characterized by progressive muscle wasting and weakness of variable distribution and severity [1]. These symptoms are caused by cycles of myofiber degeneration –regeneration, myofiber necrosis, and the initiation of the dystrophy, resulting in the general pathologies of MDs, such as increased fibrosis, appearance of smaller and split myofibers, and reduced muscle mass [2–5]. The dysferlinopathies are a group of non-lethal MDs (e.g., limb girdle MDs and Myioshi myopathy) [6–8], differing in the muscle group in which the disease phenotype initiates and present a late phenotype onset, occurring in the second or third decades of life, as opposed to other MDs such as Duchenne MD (DMD) and congenital MD (CMD).
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research