A single dose of GC301, a gene therapy candidate in the pipeline at Beijing Genecradle Therapeutics, reduced the need for regular enzyme replacement therapy (ERT) for children older than 1 year with infantile-onset Pompe disease (IOPD), new study data show. For the children in the small study…
Discussion
People often tell me they can’t imagine what it’s like to parent a child with a rare disease. I know they mean well. Sometimes they say I’m stronger than they could ever be, or call me a superhero, or wonder how I manage it all. I understand what they’re trying…
Discussion
Switching to Nexviadyme (avalglucosidase alfa) may help stabilize declining walking ability in adults with late-onset Pompe disease whose symptoms worsen despite treatment with Myozyme (alglucosidase alfa), according to a real-world study from France. Researchers found that the group’s decline in walking ability halted during the first year…
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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