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, 14 (3).

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Abstract

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 final published version of the article (version of record). It first appeared online via Future Medicine at https://doi.org/10.2217/fnl-2019-0006 - please refer to any applicable terms of use of the publisher.
Uncontrolled Keywords: drug repositioning, motor neuron, neuromuscular disorders, neuromuscular junction, skeletal muscle, spinal muscular atrophy, survival motor neurontherapies
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: 04 Oct 2019 11:23
URI: https://eprints.keele.ac.uk/id/eprint/6306

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