A Phase 2 clinical trial testing the experimental oral therapy S-606001 as an add-on treatment for adults with late-onset Pompe disease (LOPD) has begun enrolling patients, according to an announcement from the trial’s sponsor, Shionogi. “This is an important milestone for Shionogi, as we continue to expand our…
Discussion
When my child was diagnosed with Pompe disease, I realized pretty quickly that understanding the condition was just one part of what we faced. The other part was explaining it to everyone — family, friends, teachers, other parents at the playground, even strangers who noticed something and asked questions…
ABX1100, an experimental substrate reduction therapy for late-onset Pompe disease (LOPD), was well tolerated and seemed to work as designed in an early clinical trial involving nine adults with LOPD, new data show. Developer Aro Biotherapeutics noted that, in addition to meeting the study’s primary endpoints, or goals,…
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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