Whenever I see a family sitting in a waiting room, I can’t help but wonder about their story. Are they just starting out on their Pompe disease journey? Are they trying to make sense of a diagnosis they never expected, feeling lost in a world of medical terms and…
Discussion
Most adults with late-onset Pompe disease (LOPD) interviewed after participating in clinical studies of Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) found the treatment manageable, and more than half reported improvements in their quality of life, a small interview study shows. Participants also generally felt well informed about the studies…
Discussion
Every fall, I round up my kids for their flu shots. And every time, you’d think I was asking them to endure some sort of medieval torture. There’s bargaining, pleading, and all sorts of creative attempts to get out of it. No one is excited for flu shot day, but…
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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