Spinal muscular atrophy (SMA) is a congenital neuromuscular disorder characterized by
motor neuron loss, resulting in progressive weakness. SMA is notable in the health care …

Systemically low levels of survival motor neuron-1 (SMN1) protein cause spinal muscular
atrophy (SMA). α-Motor neurons of the spinal cord are considered particularly vulnerable in …

Autosomal-recessive spinal muscular atrophy (SMA) is characterized by the loss of specific
motor neurons of the spinal cord and skeletal muscle atrophy. SMA is caused by mutations …

Spinal muscular atrophy (SMA) describes a group of disorders associated with spinal motor
neuron loss. In this review we provide an update regarding the most common form of SMA …

Spinal muscular atrophy (SMA) is characterized by motor neuron loss, caused by mutations
or deletions in the ubiquitously expressed survival motor neuron 1 (SMN1) gene. We …

Of the numerous inherited diseases known to afflict the pediatric population, spinal muscular
atrophy (SMA) is among the most common. It has an incidence of approximately one in …

Spinal muscular atrophies (SMA) are a group of motor neuron diseases characterized by
degeneration of anterior horn cells of the spinal cord and by muscular atrophy. Childhood …

Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular
disease, characterized by the degeneration of lower motor neurons in the spinal cord and …

PURPOSE OF REVIEW This article provides an overview of the pathophysiology and clinical
presentations of spinal muscular atrophy (SMA) and reviews therapeutic developments …

Spinal muscular atrophy (SMA) is typically characterized as a motor neuron disease, but
extraneuronal phenotypes are present in almost every organ in severely affected patients …