Hoolachan, J, Sutton, E and Bowerman, M ORCID: https://orcid.org/0000-0002-3579-6403 (2019) Teaching an old drug new tricks: repositioning strategies for spinal muscular atrophy. Future Neurology. (In Press)

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Spinal muscular atrophy (SMA) is a childhood disorder caused by loss of the survival motor neuron (SMN) gene. Pathological hallmarks are spinal cord motor neuron death, neuromuscular junction dysfunction and muscle atrophy. The first SMN genetic therapy was recently approved and other SMN-dependent treatments are not far behind. However, not all SMA patients will reap their maximal benefit due to limited accessibility, high costs and differential effects depending on timing of administration and disease severity. The repurposing of commercially available drugs is an interesting strategy to ensure more rapid and less expensive access to new treatments. In this mini-review, we will discuss the potential and relevance of repositioning drugs currently used for neurodegenerative, neuromuscular and muscle disorders for SMA.

Item Type: Article
Additional Information: This is the accepted author manuscript (AAM). The final published version (version of record) will be available online via Future Medicine at http://www.futuremedicine.com/loi/fnl - please refer to any applicable terms of use of the publisher.
Subjects: R Medicine > RC Internal medicine > RC346 Neurology. Diseases of the nervous system, including speech disorders
Divisions: Faculty of Medicine and Health Sciences > School of Medicine
Depositing User: Symplectic
Date Deposited: 09 May 2019 13:04
Last Modified: 09 May 2019 13:08
URI: http://eprints.keele.ac.uk/id/eprint/6306

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