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Teaching an old drug new tricks: repositioning strategies for spinal muscular atrophy

Bowerman

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Authors



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.

Journal Article Type Article
Acceptance Date May 9, 2019
Publication Date Aug 22, 2019
Publicly Available Date Mar 28, 2024
Journal Future Neurology
Print ISSN 1479-6708
Publisher Future Medicine
Peer Reviewed Peer Reviewed
Volume 14
Issue 3
DOI https://doi.org/10.2217/fnl-2019-0006
Keywords drug repositioning, motor neuron, neuromuscular disorders, neuromuscular junction, skeletal muscle, spinal muscular atrophy, survival motor neurontherapies
Publisher URL https://doi.org/10.2217/fnl-2019-0006