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

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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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 ...

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 ...

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.

After hours: February 21 at 4:28:40 PM EST ...

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

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neuronal ceroid lipofuscinosis, ATP13A2-related, dog (MONDO:1012707) genetic epilepsy with febrile seizures plus spectrum (MONDO:0800489) persistent truncus arteriosus, GATA6-related, cattle ...