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2001, Contemporary neurology series, ISBN 0195133013, Volume 59., xxi, 695
Book
Annals of Neurology, ISSN 0364-5134, 01/2017, Volume 81, Issue 1, pp. 164 - 165
Journal Article
04/2019, ISBN 9783030030940, 804
An updated overview of muscle gene therapy is provided in this comprehensive reference. The test highlights important gene therapy applications, in addition to... 
Muscles-Diseases
eBook
Annals of Neurology, ISSN 0364-5134, 01/2017, Volume 81, Issue 1, pp. 164 - 165
Journal Article
Annals of Neurology, ISSN 0364-5134, 01/2017, Volume 81, Issue 1, p. 164
Journal Article
Annals of Neurology, ISSN 0364-5134, 01/2017, Volume 81, Issue 1, p. 164
Journal Article
Annals of Neurology, ISSN 0364-5134, 01/2017, Volume 81, Issue 1, pp. 164 - 165
Journal Article
Neurology, ISSN 0028-3878, 09/2019, Volume 93, Issue 10, pp. 463 - 463
Journal Article
Neurology, ISSN 0028-3878, 11/2018, Volume 91, Issue 20, pp. 909 - 910
Journal Article
Pediatric Pulmonology, ISSN 8755-6863, 02/2019, Volume 54, Issue 2, pp. 179 - 185
Background Spinal Muscular Atrophy type 1 (SMA1) is a rare genetic neuromuscular disease where 75% of SMA1 patients die/require permanent‐ventilation by 13.6... 
health outcomes | gene replacement | AVXS‐101 | gene therapy | spinal muscular atrophy | quality of life | SMA1 | AVXS-101 | DIAGNOSIS | MANAGEMENT | MODEL | CARE | CHILDREN | DELIVERY | RESPIRATORY SYSTEM | PEDIATRICS | QUALITY-OF-LIFE | NONINVASIVE VENTILATION | Original | Pcd, Pig, Nehi, Child, and Rare Diseases
Journal Article
Pediatric neurology, ISSN 0887-8994, 06/2019
Spinal muscular atrophy is a devastating neurodegenerative autosomal recessive disease that results from survival of motor neuron 1 (SMN1) gene mutation or... 
Journal Article
Annals of Neurology, ISSN 0364-5134, 02/2014, Volume 75, Issue 2, pp. 329 - 329
Journal Article
Annals of Neurology, ISSN 0364-5134, 02/2014, Volume 75, Issue 2, pp. 329 - 329
Journal Article
Annals of Neurology, ISSN 0364-5134, 02/2014, Volume 75, Issue 2, p. 329
Journal Article
Annals of Neurology, ISSN 0364-5134, 2014, Volume 75, Issue 2, p. 329
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Annals of neurology, 01/2017, Volume 81, Issue 1, p. 164
Journal Article
The New England journal of medicine, 02/2018, Volume 378, Issue 5, p. 487
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