X-linked DCM was first described in 1987 as DCM occurring in males in the teen years and early twenties, with rapid progression from congestive heart failure to death or transplantation. Dr. Jeffrey ...

DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn causes degeneration of skeletal and cardiac muscle ...

本文通过优化非人灵长类 DMD 模型的构建，开发出 Single-cut 基因疗法。该疗法能恢复 dystrophin 蛋白表达，改善肌肉病理和运动功能，且长期有效、安全性高，为 DMD 治疗带来新希望。 ### 研究背景 杜氏肌营养不良（Duchenne muscular dystrophy，DMD）是一种严重的 X 连锁 ...

Without dystrophin, your muscle cells remain unprotected ... Children who show symptoms at a younger age are at a higher risk of heart problems than those who show symptoms later.

Cardiomyopathy is a disease that weakens the heart muscle. In DMD, low levels of dystrophin protein cause the heart to grow bigger and the heart muscle to weaken. These changes make it harder for ...

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of patients in the control group.

This is due to a fault in the gene producing dystrophin. Muscles to become weaker ... ability to breathe as well as the function of the heart, as the heart is a muscle too. The missing protein ...

Elevidys was granted expanded approval by the U.S. Food and Drug Administration in 2024, with expectations high that the therapy, designed to restore a critical muscle protein called dystrophin ...

Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation ...