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A cardiac MRI helped identify heart involvement in a 16-year-old boy with juvenile Batten disease, also known as CLN3 disease, a study showed. Researchers were able to detect abnormalities in his heart’s myocardium, or muscle layer, indicative of cardiac involvement. “The clinical use of cardiac MRI should be considered…
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Theranexus and the Beyond Batten Disease Foundation (BBDF) have completed enrollment for a Phase 1/2 clinical trial testing Batten-1 — the partners’ revamped experimental therapy, solely containing miglustat — in adolescents and adults with juvenile Batten disease. This is “an important milestone in the development of Batten-1, a…
An experimental enzyme replacement therapy (ERT) — one injected directly into the brain — was found to lessen motor dysfunction and nerve cell death in mouse and sheep models of infantile Batten disease, a new study reports. Collaborations Pharmaceuticals, known as CPI, recently received nearly $3 million…
Several years after her sister’s death, columnist Laura King Edwards is learning to approach advocacy as a marathon, not a sprint.
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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