Results 131 to 140 of about 641,821 (189)
Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes. [PDF]
J Pediatr (Rio J)Santos ARBV +4 more
europepmc +1 more source
Long-term persistence, safety and effectiveness of nusinersen in spinal muscular atrophy: a population-based study. [PDF]
J NeurolAragon-Gawinska K +7 more
europepmc +1 more source
Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy. [PDF]
Pediatr PulmonolThébault C +9 more
europepmc +1 more source
Epidemiology of Spinal Muscular Atrophy in Iceland: A Population-Based Study.
NeuroepidemiologyMainka Sveindóttir AS +3 more
europepmc +1 more source
Neurologic Clinics, 2015 Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder which affects α-motor neurons in anterior horns of spinal cord resulting in progressive muscle weakness.
Stephen J Kolb, John T Kissel
exaly +5 more sources
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Seminars in Pediatric Neurology, 2021
Spinal muscular atrophy is one of the most common neuromuscular disorders of childhood and has high morbidity and mortality. Three different disease-modifying treatments were introduced in the last 4 years: nusinersen, onasemnogene abeparvovec, and risdiplam.
Stefan, Nicolau +3 more
openaire +2 more sources
Spinal muscular atrophy is one of the most common neuromuscular disorders of childhood and has high morbidity and mortality. Three different disease-modifying treatments were introduced in the last 4 years: nusinersen, onasemnogene abeparvovec, and risdiplam.
Stefan, Nicolau +3 more
openaire +2 more sources
Continuum, 2020
This article provides an overview of the pathophysiology and clinical presentations of spinal muscular atrophy (SMA) and reviews therapeutic developments, including US Food and Drug Administration (FDA)-approved gene-targeted therapies and mainstays of supportive SMA care.Over the past decades, an understanding of the role of SMN protein in the ...
Haluk, Topaloglu, Nancy, Kuntz
openaire +4 more sources
This article provides an overview of the pathophysiology and clinical presentations of spinal muscular atrophy (SMA) and reviews therapeutic developments, including US Food and Drug Administration (FDA)-approved gene-targeted therapies and mainstays of supportive SMA care.Over the past decades, an understanding of the role of SMN protein in the ...
Haluk, Topaloglu, Nancy, Kuntz
openaire +4 more sources