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…
News
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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