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Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy.
- Source :
-
Neuron [Neuron] 2018 Mar 07; Vol. 97 (5), pp. 1001-1003. - Publication Year :
- 2018
-
Abstract
- Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering new treatment options and renewed hope to SMA patients.<br /> (Copyright © 2018 Elsevier Inc. All rights reserved.)
- Subjects :
- Animals
Genetic Therapy trends
Humans
Motor Neurons pathology
Motor Neurons physiology
Randomized Controlled Trials as Topic methods
Spinal Muscular Atrophies of Childhood diagnosis
Survival of Motor Neuron 2 Protein genetics
Treatment Outcome
Genetic Therapy methods
Spinal Muscular Atrophies of Childhood genetics
Spinal Muscular Atrophies of Childhood therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1097-4199
- Volume :
- 97
- Issue :
- 5
- Database :
- MEDLINE
- Journal :
- Neuron
- Publication Type :
- Academic Journal
- Accession number :
- 29518354
- Full Text :
- https://doi.org/10.1016/j.neuron.2018.02.009