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 ...

Purpose: Late-infantile neuronal ceroid lipofuscinosis (LINCL), an autosomal recessively inherited lysosomal storage disorder characterized by autofluorescent inclusions and rapid progression of ...

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Batten disease is a rare genetic disorder affecting 3 in 100,000 babies in the U.S., causing neurodegeneration, seizures, ...

Nine-year-old Charleigh Pollock, who attends Happy Valley School, will soon lose funding for the enzyme replacement infusions ...

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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.

a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic ...

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B.C. Health Minister Josie Osborne has directed staff to investigate the case of a terminally ill Langford child who is weeks ...

These were the six commonest diagnostic groups: leukoencephalopathies (183 cases), neuronal ceroid lipofuscinoses (141 cases), mitochondrial diseases (122 cases), mucopolysaccharidoses (102 cases), ...