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 …

Recent advances in the treatment of spinal muscular atrophy (SMA) have dramatically
altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form …

Ever since loss of survival motor neuron (SMN) protein was identified as the direct cause of
the childhood inherited neurodegenerative disorder spinal muscular atrophy, significant …

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by abnormally low
cellular levels of the ubiquitous protein SMN. Traditionally, reduced levels of SMN were …

Motor neuron diseases (MNDs) are an etiologically heterogeneous group of disorders of
neurodegenerative origin, which result in degeneration of lower (LMNs) and/or upper motor …

Motor neuron diseases are neurological disorders characterized primarily by the
degeneration of spinal motor neurons, skeletal muscle atrophy, and debilitating and often …

Paucity of the survival motor neuron (SMN) protein triggers the oft-fatal infantile-onset motor
neuron disorder, spinal muscular atrophy (SMA). Augmenting the protein is one means of …

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by homozygous
mutations or deletions in the survival of motor neuron (SMN1) gene, encoding the …

Homozygous loss of SMN1 causes spinal muscular atrophy (SMA), the most common and
devastating childhood genetic motor-neuron disease. The copy gene SMN2 produces …

The inherited motor neuron disease spinal muscular atrophy (SMA) is caused by deficient
expression of survival motor neuron (SMN) protein and results in severe muscle weakness …