Spinal muscular atrophy—recent therapeutic advances for an old challenge
I Faravelli, M Nizzardo, GP Comi, S Corti - Nature Reviews Neurology, 2015 - nature.com
In the past decade, improved understanding of spinal muscular atrophy (SMA)
aetiopathogenesis has brought us to a historical turning point: we are at the verge of …
aetiopathogenesis has brought us to a historical turning point: we are at the verge of …
Spinal muscular atrophy: a timely review
SJ Kolb, JT Kissel - Archives of neurology, 2011 - jamanetwork.com
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 …
motor neurons in the anterior horn of the spinal cord and resultant weakness. The most …
Treatment advances in spinal muscular atrophy
D Bharucha-Goebel, P Kaufmann - Current Neurology and Neuroscience …, 2017 - Springer
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 …
neurons in the anterior horns of the spinal cord and brainstem that results in muscle atrophy …
New and developing therapies in spinal muscular atrophy: from genotype to phenotype to treatment and where do we stand?
TH Chen - International journal of molecular sciences, 2020 - mdpi.com
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 …
motor neuron loss, resulting in progressive weakness. SMA is notable in the health care …
Spinal muscular atrophy: going beyond the motor neuron
G Hamilton, TH Gillingwater - Trends in molecular medicine, 2013 - cell.com
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 …
cellular levels of the ubiquitous protein SMN. Traditionally, reduced levels of SMN were …
Time is motor neuron: therapeutic window and its correlation with pathogenetic mechanisms in spinal muscular atrophy
A Govoni, D Gagliardi, GP Comi, S Corti - Molecular neurobiology, 2018 - Springer
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder
characterized by the degeneration of lower motor neurons (MNs) in the spinal cord and …
characterized by the degeneration of lower motor neurons (MNs) in the spinal cord and …
In search of a cure: the development of therapeutics to alter the progression of spinal muscular atrophy
KS Ojala, EJ Reedich, CJ DiDonato, SD Meriney - Brain Sciences, 2021 - mdpi.com
Until the recent development of disease-modifying therapeutics, spinal muscular atrophy
(SMA) was considered a devastating neuromuscular disease with a poor prognosis for most …
(SMA) was considered a devastating neuromuscular disease with a poor prognosis for most …
[HTML][HTML] Spinal muscular atrophy therapeutics: where do we stand?
C d'Ydewalle, CJ Sumner - Neurotherapeutics, 2015 - Elsevier
Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder pathologically
characterized by the degeneration of motor neurons in the spinal cord and muscle atrophy …
characterized by the degeneration of motor neurons in the spinal cord and muscle atrophy …
Advances in spinal muscular atrophy therapeutics
V Parente, S Corti - Therapeutic advances in neurological …, 2018 - journals.sagepub.com
Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular
disease, characterized by the degeneration of lower motor neurons in the spinal cord and …
disease, characterized by the degeneration of lower motor neurons in the spinal cord and …