Clinical review report : indication : for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency. Cerliponase Alfa (Brineura) (Biomarin Pharmaceutical (Canada) Inc.) / Canadian Agency for Drugs and Technologies in Health.

Format:

Book

Author/Creator:

Canadian Agency for Drugs and Technologies in Health, author, issuing body.

Language:

English

Subjects (All):

Outcome assessment (Medical care).

Physical Description:

1 online resource

Edition:

Final (with redactions).

Place of Publication:

Ottawa (ON) : CADTH, 2019.

Summary:

The objective of this report was to perform a systematic review of the beneficial and harmful effects of cerliponase alfa for the treatment of pediatric patients with neuronal ceroid lipofuscinosis type 2 disease.

Contents:

Abbreviations

Executive Summary

Introduction

Results and Interpretation

Conclusions

Disease Prevalence and Incidence

Standards of Therapy

Drug

Objectives and Methods

Objectives

Methods

Results

Findings From the Literature

Included Studies

Exposure to Study Treatments

Critical Appraisal

Efficacy

Harms

Discussion

Summary of Available Evidence

Interpretation of Results

Appendix 1. Patient Input Summary

Appendix 2. Literature Search Strategy

Appendix 3. Excluded Studies

Appendix 4. Detailed Outcome Data

Appendix 5. Validity of Outcome Measures

References.

Notes:

Includes bibliographical references.

Description based on publisher supplied metadata and other sources.