A Hypothesized Therapeutic Role of (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD)

被引：0

作者：

Remmel, H. Lawrence ^{[1
,2
,3
,4
,5
,6
]}

Hammer, Sandra S. ^{[1
]}

Neff, Laurence A. ^{[7
,8
]}

Dorchies, Olivier M. ^{[7
,8
,9
]}

Scapozza, Leonardo ^{[7
,8
]}

Fischer, Dirk ^{[10
]}

Quay, Steven C. ^{[1
]}

机构：

[1] Atossa Therapeut Inc, Seattle, WA 98125 USA

[2] Univ Med Ctr Utrecht, Dept Pathol, Utrecht, Netherlands

[3] Weill Cornell Med, Childrens Canc & Blood Fdn Labs, Dept Pediat, Meyer Canc Ctr, New York, NY 10065 USA

[4] Weill Cornell Med, Childrens Canc & Blood Fdn Labs, Meyer Canc Ctr, Dept Cell & Dev Biol, New York, NY 10065 USA

[5] Weill Cornell Med, Drukier Inst Childrens Hlth, New York, NY 10065 USA

[6] Weill Cornell Med, Dept Pediat, New York, NY 10065 USA

[7] Univ Geneva, Sch Pharmaceut Sci, Geneva, Switzerland

[8] Univ Geneva, Inst Pharmaceut Sci Western Switzerland, Geneva, Switzerland

[9] Univ Grenoble Alpes, Inserm U1055, Lab Fundamental & Appl Bioenerget, Grenoble, France

[10] Univ Basel, Univ Childrens Hosp Basel UKBB, Div Pediat Neurol & Dev Med, Basel, Switzerland

来源：

DEGENERATIVE NEUROLOGICAL AND NEUROMUSCULAR DISEASE | 2025年 / 15卷

关键词：

Endoxifen; Duchenne Muscular Dystrophy; DMD carrier associated pathologies; repurposed therapy; estrogen; protein kinase C; ESTROGEN-RECEPTOR; SKELETAL-MUSCLE; DOUBLE-BLIND; MOUSE MODEL; TAMOXIFEN; BETA; REGENERATION; INHIBITION; ENDOXIFEN; SAFETY;

D O I：

10.2147/DNND.S496904

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：1 / 15

页数：15