Researchers in Taiwan have developed new molecules that can stabilize the acid alpha-glucosidase (GAA) enzyme, a strategy they say might improve the effectiveness of treatments for Pompe disease. Their study, “Harnessing polyhydroxylated pyrrolidines as a stabilizer of acid alpha-glucosidase (GAA) to enhance the efficacy of enzyme…
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FORTIS, a Phase 1/2 clinical trial of an experimental gene therapy called AT845, is due to restart in adults with late-onset Pompe disease (LOPD), thanks to a decision by the U.S. Food and Drug Administration (FDA). The trial was put on hold mid-2022 after one of its…
Researchers created and characterized an infantile-onset Pompe disease (IOPD) mouse model that carried a genetic defect commonly found among people with Southern Han Chinese ancestry, a study reported. The model recapitulated most of the signs and symptoms of IOPD and, according to researchers, is ideal to evaluate potential IOPD…
Astellas Gene Therapies has obtained the rights to develop medication that could help reduce preexisting immune responses against the viral carrier used in ATA845, its investigational gene therapy for adults with late-onset Pompe disease (LOPD). Called IdeXork, or Xork, the investigational medication was developed by Selecta Biosciences,…
MRI scans of people with classic infantile Pompe disease showed a change in the microscopic structure of certain brain regions — but no damage was seen in patients with the late-onset form of the disease, a new study reports. “We conclude that, while no deviations from typical neurodevelopment were…
In the years since his late-onset Pompe diagnosis, columnist Dwayne M. Wilson has thrown himself into raising awareness of his rare disease.
Check out Pompe Disease News‘ collection of Health Insights, where you can find more information and guidance.
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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