Neuronal Ceroid Lipofuscinosis are a group of devastating neurodegenerative lysosomal storage disorders that begin in childhood. Mutations in CLN3 gene lead to a NCL called Batten disease, characterized by the progressive ...
Jul 6, 2023
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A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects. The study, conducted ...
Sep 28, 2023
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(HealthDay)—Brineura (cerliponase alfa) has been approved by the U.S. Food and Drug Administration to treat a specific form of Batten Disease, a rare set of genetic disorders that typically begin in childhood between ages ...
Apr 28, 2017
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Professor Stéphane Lefrançois, a researcher at the Institut National de la Recherche Scientifique (INRS), is working on Batten disease, a neurodegenerative genetic disease that primarily affects children. His research focuses ...
Feb 28, 2020
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