Mouse experiments with a gene therapy for juvenile Batten disease indicate that lower activation of the mutated CLN3 gene are more beneficial than high levels. While the research team was the first to show that the specific gene delivery method can be used for treating Batten disease, their findings illustrate…
The Batten Disease Support and Research Association (BDSRA) will hold its 2017 Family Conference in July, bringing together patients and families touched by Batten disease. The on July 6–9 event will take place at the Wyndham Grand Pittsburgh Downtown. The conference joins family members, children and researchers to…
Abeona Therapeutics‘ ABO-201 reduced nerve inflammation and improved movement in mice with juvenile Batten disease (JBD), according to a preclinical trial study. Scientists at Abeona designed ABO-201 to overcome the effects of the CLN3 genetic mutation that is responsible for the disease. A single injection led to the correct form of the gene being delivered…
Patients with a specific type of Batten disease now have an approved treatment, with the U.S. Food and Drug Administration’s (FDA) recent approval of Brineura (cerliponase alfa) for use in late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is an enzyme replacement therapy, providing CLN2 patients with a lab-produced…
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