Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
| العنوان: | Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 |
|---|---|
| المؤلفون: | Grace Lewis, Bernard Kim, Stephanie L Conway-Allen, Amanda M. Morrill |
| المصدر: | Journal of Child Neurology. 35:348-353 |
| بيانات النشر: | SAGE Publications, 2019. |
| سنة النشر: | 2019 |
| مصطلحات موضوعية: | 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 |
| الوصف: | 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. |
| تدمد: | 1708-8283 0883-0738 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b72e66027adcb81bc6162e42928db878Test https://doi.org/10.1177/0883073819895694Test |
| حقوق: | CLOSED |
| رقم الانضمام: | edsair.doi.dedup.....b72e66027adcb81bc6162e42928db878 |
| قاعدة البيانات: | OpenAIRE |
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