The term “neurodegenerative diseases”(NDs) identifies a group of heterogeneous diseases
characterized by progressive loss of selectively vulnerable populations of neurons, which …

Only in recent years, thanks to a precision medicine-based approach, have treatments
tailored to the sex of each patient emerged in clinical trials. In this regard, both striated …

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder leading to
paralysis, muscle atrophy, and death. Significant advances in antisense oligonucleotide …

Spinal muscular atrophy (SMA) is one of the major genetic disorders associated with infant
mortality. More than 90% of cases of SMA result from deletions of or mutations in the …

Genetic therapy has changed the prognosis of hereditary proximal spinal muscular atrophy,
although treatment efficacy has been variable. There is a clear need for deeper …

Background Natural history of spinal muscular atrophy (SMA) in adult age has not been fully
elucidated yet, including factors predicting disease progression and response to treatments …

Survival motor neuron (SMN) protein deficiency results in loss of alpha motor neurons and
subsequent muscle atrophy in patients with spinal muscular atrophy (SMA). Reactive …

The motor cortex is crucial for the voluntary control of skilled movement in mammals and is
topographically organized into representations of the body (motor maps). Intracortical …

Objectives The motor neuron survival protein, which is deficient in spinal muscular atrophy
(SMA), performs numerous cellular functions. Currently, SMA is believed to be a multi-organ …

Spinal muscular atrophy (SMA) is a monogenic neuromuscular disease caused by low
levels of the Survival Motor Neuron (SMN) protein. Motor neuron degeneration is the central …