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CADTH Canadian Drug Expert Committee Recommendation: Cerliponase Alfa (Brineura — Biomarin Pharmaceutical [Canada] Inc.): Indication: For the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2019 May.
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CADTH Canadian Drug Expert Committee Recommendation: Nitisinone (Nitisinone Tablets — Cycle Pharmaceuticals Ltd.): Indication: The treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Aug.
CADTH Canadian Drug Expert Committee Recommendation: Migalastat (Galafold — Amicus Therapeutics): Indication: Fabry Disease [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Jan.
CADTH Canadian Drug Expert Committee Recommendation: Sebelipase alfa (Kanuma — Alexion Pharmaceuticals, Inc.): Indication: Lysosomal acid lipase deficiency [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Sep 28.
CADTH Canadian Drug Expert Committee Recommendation: Nitisinone (MDK-Nitisinone — MendeliKABs Inc.): Indication: The treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Apr.
CADTH Canadian Drug Expert Committee Recommendation: Eliglustat (Cerdelga — Sanofi Genzyme): Indication: Type 1 Gaucher disease [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2017 Jul.
CADTH Canadian Drug Expert Committee Recommendation: Nitisinone (Orfadin — Sobi Canada Inc.): Indication: The treatment of adult and pediatric patients with hereditary tyrosinemia type-1 in combination with a dietary restriction of tyrosine and phenylalanine [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb.
CADTH Canadian Drug Expert Committee Final Recommendation Sapropterin — Resubmission: (Kuvan — BioMarin Pharmaceuticals Canada Inc.): Indication: To reduce blood phenylalanine levels in patients with hyperphenylalaninemia due to BH4-responsive phenylketonuria [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2016 Oct 26.
CADTH Canadian Drug Expert Committee Recommendation: Cysteamine Delayed-Release (Procysbi — Horizon Pharma Ireland Ltd.): Indication: Nephropathic cystinosis [Internet].
CADTH Canadian Drug Expert Committee Recommendation: Patisiran (Onpattro — Alnylam Netherlands BV): Indication: For the treatment of polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2019 Jul.
CADTH Canadian Drug Expert Committee Recommendation: Cysteamine Ophthalmic Solution (Cystadrops — Recordati Rare Diseases Canada Inc.): Indication: Treatment of corneal cystine crystal deposits (CCCDs) in adults and children from 2 years of age with cystinosis [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2019 Jun.
Triheptanoin (Dojolvi): CADTH Reimbursement Recommendation: Indication: As a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD) [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2022 Feb.
Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action.
National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Population Health and Public Health Practice; Committee on Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action; Martinez RM, Osei-Anto HA, McCormick M, editors.
Washington (DC): National Academies Press (US); 2020 Sep 10.
Pegvaliase (Palynziq): CADTH Reimbursement Recommendation: Indication: For the treatment of patients with phenylketonuria aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 µmol/L) on existing management [Internet].
Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2022 Nov.
PDQ Cancer Information Summaries [Internet].
Bethesda (MD): National Cancer Institute (US); 2002-.
Baby-Friendly Hospital Initiative: Revised, Updated and Expanded for Integrated Care.
Geneva: World Health Organization; 2009.
Genetic Susceptibility to Breast and Ovarian Cancer: Assessment, Counseling and Testing Guidelines.
Bethesda (MD): American College of Medical Genetics; 1999.
Guideline: Updates on Paediatric Emergency Triage, Assessment and Treatment: Care of Critically-Ill Children.
Geneva: World Health Organization; 2016.
Coronavirus Disease 2019 (COVID-19) Treatment Guidelines [Internet].
Bethesda (MD): National Institutes of Health (US); 2021 Apr 21-2024 Feb 29.
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