Background Spinal muscular atrophy (SMA) is a genetic motor neuron disease related to
deletions in the SMN1 gene. There is mounting evidence that the disease is not restricted to …

Spinal muscular atrophy (SMA) type III and IV are autosomal recessive, slowly progressive
lower motor neuron syndromes. Nevertheless, wider cerebral involvement has been …

Spinalmuscular atrophy (SMA) is a neuromuscular disease that affects as many as 1 in 6000
individuals at birth, making it the leading genetic cause of infant mortality. A growing number …

Background: 5q Spinal Muscular Atrophy (SMA) is a prototypical lower motor neuron
disorder. However, the characteristic early motor impairment raises the question on the …

Spinal muscular atrophy (SMA) is a neurogenetic autosomal recessive disorder
characterized by degeneration of lower motor neurons. The validation of appropriate animal …

Spinal muscular atrophy (SMA) is a common autosomal recessive neuromuscular disorder
that is characterized by degeneration of the anterior horn cells of the spinal cord, which …

Background Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular
disorder caused by homozygous deletion or intragenic mutation of the SMN1 gene. It is well …

Understanding the events that are responsible for a disease is mandatory for setting up a
therapeutic strategy. Although spinal muscular atrophy (SMA) is considered a rare …

Spinal muscular atrophy (SMA) is an autosomal recessive inherited disorder caused by
alterations in the survival motor neuron I (SMN1) gene. SMA patients are classified as type I …

Background Spinal muscular atrophy (SMA) is a rare autosomal recessive hereditary
neuromuscular disease caused by survival motor neuron 1 (SMN1) gene deletion or …