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Did you mean: ( alfa)?
(cerliponase alfa)
10 results
  • Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses. [Journal Article]
    CNS Drugs 2019; 33(4):315-325Kohlschütter A, Schulz A, … Storch S
  • The neuronal ceroid lipofuscinoses comprise a group of neurodegenerative lysosomal storage disorders caused by mutations in at least 13 different genes and primarily affect the brain and the retina of children or young adults. The disorders are characterized by progressive neurological deterioration with dementia, epilepsy, loss of vision, motor disturbances, and early death. While various therap…
  • Therapeutic landscape for Batten disease: current treatments and future prospects. [Review]
    Nat Rev Neurol 2019; 15(3):161-178Johnson TB, Cain JT, … Weimer JM
  • Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of devastating lysosomal storage disorders that collectively represent the most common inherited paediatric neurodegenerative disorders worldwide. Batten disease can result from mutations in 1 of 13 genes. These mutations lead to a group of diseases with loosely overlapping symptoms and pathology. Phenotypically, p…
  • Study of Intraventricular Cerliponase Alfa for CLN2 Disease. [Clinical Trial]
    N Engl J Med 2018; 378(20):1898-1907Schulz A, Ajayi T, … CLN2 Study Group
  • CONCLUSIONS: Intraventricular infusion of cerliponase alfa in patients with CLN2 disease resulted in less decline in motor and language function than that in historical controls. Serious adverse events included failure of the intraventricular device and device-related infections. (Funded by BioMarin Pharmaceutical and others; CLN2 numbers, NCT01907087 and NCT02485899 .).
  • Cerliponase Alfa: First Global Approval. [Review]
    Drugs 2017; 77(11):1247-1249Markham A
  • Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and earl…
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