When my child was diagnosed with Pompe disease, my whole world changed in an instant. Suddenly, I wasn’t just a parent; I had to learn how to be a researcher, a scheduler, an insurance negotiator, a medication manager, and, most of all, an advocate for my child. I always…
Avenue Therapeutics has secured rights to develop clenbuterol, an experimental add-on treatment for Pompe disease that has already shown promise in early clinical testing. The company announced it has entered into an exclusive worldwide license agreement with Duke University for patents and technical expertise related to the experimental…
A single dose of a specialized gene therapy has successfully normalized muscle function for more than a year in a mouse model of Pompe disease. By using “data-mined” genetic sequences designed to supercharge enzyme production specifically in muscle cells, researchers achieved protein levels up to 30 times higher than…
Becoming educated and informed about Pompe is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnist’s words of wisdom to help you along on your Pompe journey.
There is no cure for Pompe yet, but there are treatments that can help manage the disease. Learn more about current therapeutic approaches here.
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