Until the recent development of disease-modifying therapeutics, spinal muscular atrophy
(SMA) was considered a devastating neuromuscular disease with a poor prognosis for most …

Spinal muscular atrophy (SMA) is a hereditary neurodegenerative disease with severity
ranging from progressive infantile paralysis and premature death (type I) to limited motor …

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 …

Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by loss of
motor neurons in the anterior horn of the spinal cord and resultant weakness. The most …

Spinal muscular atrophy (SMA) is a potentially devastating and lethal neuromuscular
disease frequently manifesting in infancy and childhood. The discovery of the underlying …

Twenty-five years ago, the underlying genetic cause for one of the most common and
devastating inherited diseases in humans, spinal muscular atrophy (SMA), was identified …

Abstract Purpose of Review Spinal muscular atrophy (SMA) is a genetic disorder of motor
neurons in the anterior horns of the spinal cord and brainstem that results in muscle atrophy …

Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by
deletion or mutation of SMN1. Four subtypes exist, characterized by different clinical …

Spinal muscular atrophy is an autosomal recessive neurodegenerative disorder
characterized by progressive muscle wasting and loss of muscle function due to severe …

Introduction Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative
neuromuscular disease that presents primarily in children. Abnormalities in the SMN1 gene …