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Amicus Therapeutics announced it will discontinue its investigational gene therapy program of AT-GTX-501 for late infantile neuronal ceroid lipofuscinosis 6 disease, also known as CLN6 Batten disease. The company is, however, advancing its gene therapy program for juvenile Batten disease, also known as CLN3 disease, according to a press release.
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Mutations in the CLN6 gene — which have been associated with different forms of Batten disease — may lead to the development of an atypical form of juvenile Batten that does not cause vision loss, according to a study reporting the cases of three patients from two Greek-Cypriot…
With approval from Health Canada, Taysha Gene Therapies will begin a Phase 1/2 clinical trial testing TSHA-118, its investigational gene therapy for CLN1 disease, also known as infantile Batten disease. This first trial will launch at Queen’s University, in Ontario, under the coordination of Jagdeep Walia, MD, with…
Prior to ankle surgery, columnist Laura King Edwards draws courage from her sister, Taylor, whose mobility was limited due to Batten disease.
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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