When my daughter Caroline was diagnosed with Pompe disease, I knew life would look different. I figured our routines would change, our priorities would shift, and our days would suddenly be filled with appointments, infusions, and medical conversations I never imagined I’d have. What I didn’t expect was how…
A middle-aged woman with late-onset Pompe disease (LOPD) developed damage to her sensory nerve cells after receiving the gene therapy candidate AT845 — in development as a Pompe disease treatment — in a clinical trial, according to a new case report. While the woman’s symptoms, particularly reduced sensations…
Discussion
When people hear about in vitro fertilization (IVF) after a rare disease diagnosis, they probably think the hardest part is either the cost or the medical side of things. Those parts are definitely overwhelming. But for me, the hardest part wasn’t the science; it was the question that kept circling in…
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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