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…
The first patient has been dosed in a Phase 1/2 clinical trial testing AB-1009, Askbio’s investigational gene therapy for late-onset Pompe disease (LOPD), the company said. The trial, PROGRESS-GT LOPD (NCT07282847), primarily aims to assess the therapy’s safety in up to 12 adults with LOPD, aged 18 and…
Heart problems in late-onset Pompe disease appear milder and less specific than in the disease’s infantile-onset form, a review study found. While heart scans can detect these changes, further research is needed to fully understand them, according to the researchers, who reviewed data from 11 previous studies. “Knowledge of…
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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