Spinal muscular atrophy — insights and challenges in the treatment era
Spinal muscular atrophy — insights and challenges in the treatment era
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Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publication information
Publisher
London: Nature Publishing Group UK
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Scope and Contents
Contents
Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease caused by deletion or mutation of
SMN1
. Four subtypes exist, characterized by different clinical severities. New therapeutic approaches have become available in the past few years, dramatically changing the natural history of all SMA subtypes, including substantial...
Alternative Titles
Full title
Spinal muscular atrophy — insights and challenges in the treatment era
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TN_cdi_proquest_miscellaneous_2451860684
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2451860684
Other Identifiers
ISSN
1759-4758
E-ISSN
1759-4766
DOI
10.1038/s41582-020-00413-4
