Background: LAMA2-muscular dystrophy (LAMA2-MD) is an autosomal recessive disease, and the most common form of congenital muscular (CMD). Most patients develop a disease characterized by inability to achieve walking capacity, multiple joint deformities, respiratory insufficiency, some degree dysphagia. However, there gravity spectrum, never sitting position, while others can walk unassisted. Th...

BACKGROUND The demographic development with an ageing population is predicted to be the next global public health challenge. Advances in medicine and the socioeconomic development have reduced mortality and morbidity due to infectious conditions and non-communicable diseases. The increase in longevity will not be restricted to healthy people. OBJECTIVE To understand how people with muscular d...

neurodegenerative disorders such as huntingtons disease, alzheimers disease, parkinsons disease, amyotrophic lateral sclerosis, spinal muscular atrophy, friedreichs ataxia, and others are multi-factorial illnesses in which many pathways (still poorly understood) act serially and in parallel to give a determined pathologic phenotype. thus, presently there are no effective cures for these disease...

spinal muscular atrophies (smas) represent a rare group of inherited disorders that cause progressive degeneration of the anterior horn cells of the spinal cord. the exact cause of the degeneration is unknown. loss of these cells results in a progressive lower motor neuron disease that has no sensory involvement and that is manifested as hypotonia, weakness, and progressive paralysis. kugelberg...

how to cite this article: karimzadeh p, ghazavi a. comparison of deflazacort and prednisone in duchenne muscular dystrophy. iranianjournal of child neurology 2012;6(1):5-12. objective duchenne muscular dystrophy (dmd) is a degenerative disease that usually becomes clinically detectable in childhood as progressive proximal weakness. no cure is yet available for dmd, but the use of steroids impro...

Abstract Muscular dystrophies are devastating and untreatable genetic diseases leading to progressive muscle degeneration weakness. The expanding landscape of CRISPR-Cas-based genome editing tools allows the in situ repair many disease-causing mutations patient cells an unprecedented manner. Here, I discuss recent advances challenges for using gene edited stem autologous cell replacement therap...