A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD)
Publication date
2025-03-15
Authors
Remmel, H Lawrence
Hammer, Sandra S
Neff, Laurence A
Dorchies, Olivier M
Scapozza, Leonardo
Fischer, Dirk
Quay, Steven C
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Article
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cc_by_nc
Abstract
Duchenne Muscular Dystrophy (DMD) is an inherited, X-linked disorder that is progressive, debilitating, and ultimately fatal. The current therapeutic landscape offers no cures, but does include palliative treatments that delay disease progression, and there is progress on genetic therapies that have the promise to be curative. There is much room for new therapies, and foundational work with the estrogen receptor modulator tamoxifen suggests the potential of a unique spectrum of therapeutic benefit from endoxifen, a metabolite of tamoxifen. Here we describe the potential for this new DMD therapy in the context of the overall DMD therapeutic landscape.
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Journal Article
Citation
Remmel, H L, Hammer, S S, Neff, L A, Dorchies, O M, Scapozza, L, Fischer, D & Quay, S C 2025, 'A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD)', Degenerative neurological and neuromuscular disease, vol. 15, pp. 1-15. https://doi.org/10.2147/DNND.S496904