The genetically-based therapy of spinal muscular atrophy (5qSMA) has been a pioneer in
the development of therapies for hereditary neuromuscular diseases. Within a few years, the …

In the past decade major advances have been made in the treatment of spinal muscular
atrophy, a devastating, progressive motor neuron disease caused by a deficiency in the …

Background Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor
neuron disease with an onset during infancy that results in failure to achieve motor …

Spinal muscular atrophy (SMA) used to be one of the most common genetic causes of infant
mortality. New disease modifying treatments have changed the disease trajectories and …

Spinal muscular atrophy (SMA) type 1—the leading genetic cause of infant mortality—results
from an absence of functional copies of the SMN1 gene, which encodes survival motor …

Infantile SMA is a neuromuscular disease caused by the motor neuron degeneration,
depending on the age of appearance of clinical signs and the evolution of the disease, three …

One important unanswered question is whether combining therapies that augment SMN
expression can provide greater benefits than the individual therapies alone. Combination …

Background and Objectives Spinal muscular atrophy (SMA) is a neurodegenerative disorder
manifesting with progressive muscle weakness and atrophy. SMA type 1 used to be fatal …

Spinal muscular atrophy (SMA) is the most common lethal recessive disease in childhood,
and there is currently no effective treatment to halt disease progression. The translation of …

Spinal muscular atrophy (SMA) is a serious neuromuscular disorder characterised by motor
neuron degen eration. 1 About 95% of cases of SMA involve homo zygous deletion of the …