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Animal models predicted dosing of the enzyme replacement therapy Brineura (cerliponase alfa) for patients with late infantile Batten disease, also called CLN2 disease, a study showed. Scientists said this approach also may be applied to predict cross-species dosing for other similar medicines. “Overall, the approach described here provides…
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The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
A new eye exam proved useful for testing vision in children with CLN2 disease — also known as late-infantile Batten disease — who took part in a pilot study, its developer, Objective Acuity (OAL), announced. Called threshold visual acuity test, the exam appears to be promising for…
The Batten disease community lacks the clinical management guidance needed to improve quality of life, columnist Laura King Edwards writes.
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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