Spark Therapeutics launches gene therapy clinical trial for late-onset Pompe disease – News-Medical.Net

Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in various tissues, in particular the heart, the muscles, the liver and the nervous system.
In patients living with late-onset Pompe disease the respiratory system and mobility are most frequently the most affected by the disease, often requiring the use of a wheelchair and respiratory assistance, and life expectancy can be…

Click here to view the original article.