TY - JOUR
T1 - A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD)
AU - Remmel, H Lawrence
AU - Hammer, Sandra S
AU - Neff, Laurence A
AU - Dorchies, Olivier M
AU - Scapozza, Leonardo
AU - Fischer, Dirk
AU - Quay, Steven C
N1 - © 2025 Remmel et al.
PY - 2025/3/15
Y1 - 2025/3/15
N2 - 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.
AB - 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.
U2 - 10.2147/DNND.S496904
DO - 10.2147/DNND.S496904
M3 - Article
C2 - 40124418
SN - 1179-9900
VL - 15
SP - 1
EP - 15
JO - Degenerative neurological and neuromuscular disease
JF - Degenerative neurological and neuromuscular disease
ER -