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|a CADTH Canadian Drug Expert Committee recommendation: Cerliponase Alfa (Brineura -- Biomarin Pharmaceutical [Canada] Inc.)
|h Elektronische Ressource
|b indication : for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency
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|a Cerliponase Alfa (Brineura -- Biomarin Pharmaceutical [Canada] Inc.)
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|a Drug reimbursement recommendation Cerliponase Alfa (Brineura)
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|a Version 1.0. (final)
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|a Ottawa (ON)
|b CADTH
|c May 2019, 2019
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|a 1 PDF file (11 pages)
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|a Insurance, Health, Reimbursement
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|a Neuronal Ceroid-Lipofuscinoses / drug therapy
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|a Dipeptidyl-Peptidases and Tripeptidyl-Peptidases / therapeutic use
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|a Canada
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|a Dipeptidyl-Peptidases and Tripeptidyl-Peptidases / economics
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|a Cost-Benefit Analysis
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|a CADTH Canadian Drug Expert Committee
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|a eng
|2 ISO 639-2
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|b NCBI
|a National Center for Biotechnology Information
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|a CADTH common drug review
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|u https://www.ncbi.nlm.nih.gov/books/NBK543392
|3 Volltext
|n NLM Bookshelf Books
|3 Volltext
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|a 610
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|a 330
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|a The CADTH Canadian Drug Expert Committee (CDEC) recommends that cerliponase alfa be reimbursed for the treatment of CLN2 disease, also known as TPP1 deficiency, if the following conditions are met: Conditions for Reimbursement. Initiation Criteria. 1. A confirmed diagnosis of CLN2 disease based on TPP1 enzyme activity and CLN2 genotype analysis. 2. Patient who meets all of the following: 2.1. has a minimum score of ≥ 1 in each of the motor and the language domains of the CLN2 Clinical Rating Scale. 2.2. has an aggregate motor-language score of ≥ 3 on the CLN2 Clinical Rating Scale. Discontinuation Criteria: 1. Patients must be assessed every 24 weeks for changes in motor and language function using the CLN2 Clinical Rating Scale. 2. Treatment must be discontinued if: 2.1. there is a reduction of ≥ 2 points in the aggregate motor-language score of the CLN2 Clinical Rating Scale that is maintained over any two consecutive 24-week assessments; or 2.2. the aggregate motor-language score of the CLN2 Clinical Rating Scale reaches zero at two consecutive 24-week assessments
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