When you raise a child with a rare disease, it is easy to become consumed by your own version of that disease. You learn your child’s numbers, memorize the medical terminology, track appointments, and pay attention to every change that might mean something. You become familiar with the research, the…
Discussion
Using a short course of low-dose methotrexate, an immunosuppressive agent, alongside enzyme replacement therapy (ERT) in people with Pompe disease may help prevent the formation of antibodies that can reduce treatment effectiveness. That’s the key finding of a new study by Duke University researchers, who found that…
Discussion
Long-term treatment with the combination therapy Pombiliti + Opfolda (cipaglucosidase alfa and miglustat) helps people with late-onset Pompe disease preserve their walking ability and lung function for at least five years, new clinical trial data show. Biomarin Pharmaceutical, which markets Pombiliti + Opfolda after acquiring 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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