Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
| Autor: | Grace Lewis, Bernard Kim, Stephanie L Conway-Allen, Amanda M. Morrill |
|---|---|
| Rok vydání: | 2019 |
| Předmět: |
0301 basic medicine
medicine.medical_specialty Batten disease Cerliponase alfa Gastroenterology Tripeptidyl peptidase 03 medical and health sciences 0302 clinical medicine Neuronal Ceroid-Lipofuscinoses Internal medicine medicine Humans Enzyme Replacement Therapy Dipeptidyl-Peptidases and Tripeptidyl-Peptidases Adverse effect Tripeptidyl-Peptidase 1 business.industry virus diseases Enzyme replacement therapy medicine.disease Recombinant Proteins Neuronal Ceroid Lipofuscinosis Type 2 Treatment Outcome 030104 developmental biology Pediatrics Perinatology and Child Health Disease Progression Vomiting Neuronal ceroid lipofuscinosis Neurology (clinical) medicine.symptom business 030217 neurology & neurosurgery |
| Zdroj: | Journal of Child Neurology. 35:348-353 |
| ISSN: | 1708-8283 0883-0738 |
| DOI: | 10.1177/0883073819895694 |
| Popis: | The objective of this review is to summarize the pharmacology, efficacy, and safety of cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Cerliponase alfa is recombinant human tripeptidyl peptidase 1 enzyme replacement therapy. A phase 1/2 trial established the efficacy and safety of cerliponase alfa for treatment of neuronal ceroid lipofuscinosis type 2. Treatment with intracerebroventricular cerliponase alfa resulted in slower decline of motor and language functions compared with natural history controls. Common adverse events include convulsions, electrocardiography abnormalities, pyrexia, vomiting, and upper respiratory tract infections. Intracerebroventricular device–related adverse events also occur. Cerliponase alfa is the first therapy for neuronal ceroid lipofuscinosis type 2 that targets the disease etiology. Cerliponase alfa is effective in delaying the progression of motor language decline for patients with neuronal ceroid lipofuscinosis type 2. |
| Databáze: | OpenAIRE |
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