Year: 2025
Type: article
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. Keywords: Endoxifen, Duchenne Muscular Dystrophy, DMD carrier associated pathologies, repurposed therapy, estrogen, protein kinase C
DOI: 10.2147/dnnd.s496904
Institutions: University Medical Center Utrecht, Children's Cancer and Blood Foundation, Atossa Therapeutics (United States), Weill Cornell Medicine, University of Geneva, Inserm, Laboratory of Fundamental and Applied Bioenergetics, Université Grenoble Alpes, University of Basel, University Children’s Hospital Basel
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: diamond
OpenAlex ID: W4408451829