Pompe disease will always be a form of muscular dystrophy that deteriorates the muscles throughout the body causing muscle weakness. The science of it will never change, but the treatment is something ...
HealthSpotlight offers in-depth insights and expert analysis on the latest research, trends, and resources related to Pompe Disease. Our weekly newsletter covers essential topics, including ...
I have been living with Pompe disease for almost 34 years. Because this is a genetic disorder that I was born with, I have observed a continuous change throughout my entire life. From a weird-looking ...
Koeberl’s research focuses on using adeno-associated virus (AAV) vectors to insert DNA or RNA into targeted cells where it can repair faulty genes that cause disease. Pompe disease is triggered by ...
HealthSpotlight offers in-depth insights and expert analysis on the latest research, trends, and resources related to Sickle Cell Disease. Our weekly newsletter covers essential topics ...
Treating chronic disease has proven difficult because there is not one simple cause, like a single gene mutation, that a treatment could target. At least, that's how it has appeared to scientists ...