The first day of school is usually about freshly sharpened pencils, new backpacks, and figuring out where to go after lunch. But for my daughter Caroline, the first day this year was about feeling anxious — not just about the first day, but about whether she would make it through…
Discussion
Home infusions of enzyme replacement therapy (ERT) are safe for children with Pompe disease after an initial period of hospital-based treatment, a Dutch study spanning more than 20 years found. Infusion-associated reactions occurred in fewer than 5% of nearly 12,000 infusions, and most were mild. About half of…
Discussion
When people meet my daughter Caroline, they sometimes tell me how lucky she is. They say she is lucky to have such a supportive family. Lucky to have parents who advocate for her. Lucky to have access to the treatment she needs. Lucky to have a sister who looks…
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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