A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD)
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.
Authors
- Steven C. Quay (ORCID: https://orcid.org/0000-0002-0363-7651)
- L. Neff (ORCID: https://orcid.org/0000-0002-4472-8453)
- O.M. Dorchies (ORCID: https://orcid.org/0000-0002-6665-7887)
- H. Lawrence Remmel (ORCID: https://orcid.org/0000-0002-5742-829X)
- Léonardo Scapozza (ORCID: https://orcid.org/0000-0003-1079-648X)
- Dirk Fischer (ORCID: https://orcid.org/0000-0003-0707-9201)
- Sandra S. Hammer (ORCID: https://orcid.org/0009-0003-7760-4582)
Institutions
- University of Geneva (CH)
- Inserm (FR)
- HES-SO University of Applied Sciences and Arts Western Switzerland (CH)
- Cornell University (US)
- University Medical Center Utrecht (NL)
- Children's Cancer and Blood Foundation (US)
- University Children’s Hospital Basel (CH)
- Atossa Therapeutics (United States) (US)
- Laboratory of Fundamental and Applied Bioenergetics (FR)
- Weill Cornell Medicine (US)
- Université Grenoble Alpes (FR)
Publication Details
- Journal
- Degenerative Neurological and Neuromuscular Disease
- Published
- 2025-03-01
- DOI
- https://doi.org/10.2147/dnnd.s496904
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00