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…
Discussion
Talking about mental health isn’t the easiest thing to do. Sharing our feelings can be uncomfortable, and asking for help may seem like exposing a part of ourselves we’d rather keep hidden. I’d always shared my feelings openly and leaned on family and friends. For a long time, I thought…
ABX1100, an experimental substrate reduction therapy being developed by Aro Biotherapeutics, showed strong and sustained biological activity and early signs of clinical benefit in people with late-onset Pompe disease (LOPD), according to preliminary trial results. The therapy was also well tolerated. The preliminary results are based on the…
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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