Experimental Gene Therapy Showed Promising Safety and Efficacy in Primate Study of Late-Infantile Batten Disease
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Experimental Gene Therapy Showed Promising Safety and Efficacy in Primate Study of Late-Infantile Batten Disease

Children with late-infantile onset Batten disease (CLN2) may be treated with an enzyme replacement therapy (ERT). However, this therapy cannot reverse the damage or progression children have already experienced. Researchers…

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