It’s been over six years since my son Cayden was born with infantile-onset Pompe disease. Today, I consider myself to be an experienced parent when it comes to this disease. However, I’ll never forget what it felt like to hear the news of Cayden’s diagnosis. It’s almost an indescribable…
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Aro Biotherapeutics is seeking adults with late-onset Pompe disease (LOPD) to participate in a Phase 1b trial testing ABX1100, the company’s experimental substrate reduction treatment, at a site in Canada. “We anticipate dosing the first patient with Pompe disease in the Phase 1b study before the end of 2024,”…
Myozyme (alglucosidase alfa), sold as Lumizyme in the U.S., eased breathing but did not improve motor function of the arms and legs in an Iranian woman with late-onset Pompe disease who went undiagnosed for many years, according to a case report. The researchers say this case highlights 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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