The NCLs (neuronal ceroid lipofuscinosis) are pediatric neurodegenerative disorders. The nine clinical variants are caused by mutations in different genes (CLN1–CLN9). Six of the genes are ...
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Fatal neurodegenerative disease in kids also affects the bowel—gene therapy may helpAlso known as neuronal ceroid lipofuscinosis, the disease is named after the accumulated material inside the cells. Not having these enzymes causes progressive brain damage that leads to death.
Galvin, N., Vogler, C., Levy, B., Kovacs, A., Griffey, M., Sands, M.S. A murine model of infantile neuronal ceroid lipofuscinosis - Ultrastructural evaluation of ...
NICE had been assessing Brineura (cerliponase alfa) for children with neuronal ceroid lipofuscinosis type 2 (CLN2), a rare inherited condition affecting between one and six babies each year in the UK.
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