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Gene therapy was able to reduce the signs and symptoms of late infantile Batten disease in a mouse model carrying a common disease-causing mutation, a study shows. These findings support Regenxbio’s ongoing gene therapy program, which includes RGX-181, currently being tested as part of a single-patient study…
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Changes in the levels of certain metabolites in the brain among people with juvenile Batten disease, also called CLN3 disease, are significantly associated with multiple measures of disease severity, a study showed. Age accounted for many of these metabolite changes. Still, the levels of some brain metabolites — the…
Columnist Laura King Edwards pays tribute to her mother’s love and fierce determination after Laura’s late sister was diagnosed with Batten.
Becoming educated and informed about Batten disease 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 columnists’ words of wisdom to help you along on your journey.
There is no cure for Batten disease yet, but there are treatments that can help manage the disease. Learn more about the approved and experimental therapies here.
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