Scoto, M;
Finkel, R;
Mercuri, E;
Muntoni, F;
(2018)
Genetic therapies for inherited neuromuscular disorders.
The Lancet Child & Adolescent Health
, 2
(8)
pp. 600-609.
10.1016/S2352-4642(18)30140-8.
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Muntoni_LANCET. GENETIC THERAPIES FOR INHERITED NM DISORDERS II review Final.pdf - Accepted Version Download (1MB) | Preview |
Abstract
Inherited neuromuscular disorders encompass a broad group of genetic conditions, and the discovery of these underlying genes has expanded greatly in the past three decades. The discovery of such genes has enabled more precise diagnosis of these disorders and the development of specific therapeutic approaches that target the genetic basis and pathophysiological pathways. Such translational research has led to the approval of two genetic therapies by the US Food and Drug Administration: eteplirsen for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy, which are both antisense oligonucleotides that modify pre-mRNA splicing. In this Review we aim to discuss new genetic therapies and ongoing clinical trials for Duchenne muscular dystrophy, spinal muscular atrophy, and other less common childhood neuromuscular disorders.
| Type: | Article |
|---|---|
| Title: | Genetic therapies for inherited neuromuscular disorders |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/S2352-4642(18)30140-8 |
| Publisher version: | http://dx.doi.org/10.1016/S2352-4642(18)30140-8 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10055445 |
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