资讯

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...
None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of ...
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 ...
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.
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 ...
Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...