Betaine anhydrous in homocystinuria: results from the RoCH registry

Background The Registry of Adult and Paediatric Patients Treated with Cystadane® – Homocystinuria (RoCH) is a non-interventional, observational, multi-centre, post-authorization safety study that aimed to identify safety of betaine anhydrous (Cystadane®) in the treatment of patients with inborn erro...

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Bibliographic Details
Published in:	Orphanet journal of rare diseases Vol. 14; no. 1; pp. 66 - 10
Main Authors:	Valayannopoulos, Vassili, Schiff, Manuel, Guffon, Nathalie, Nadjar, Yann, García-Cazorla, Angels, Martinez-Pardo Casanova, Mercedes, Cano, Aline, Couce, Maria L., Dalmau, Jaime, Peña-Quintana, Luis, Rigalleau, Vincent, Touati, Guy, Aldamiz-Echevarria, Luis, Cathebras, Pascal, Eyer, Didier, Brunet, Dominique, Damaj, Léna, Dobbelaere, Dries, Gay, Claire, Hiéronimus, Sylvie, Levrat, Virginie, Maillot, François
Format:	Journal Article
Language:	English
Published:	London BioMed Central 14.03.2019 BioMed Central Ltd Springer Nature B.V BMC
Subjects:	Adolescent Adult Amino acids Betaine Betaine - administration & dosage Betaine - adverse effects Betaine anhydrous Care and treatment Child Child, Preschool Defects Edema Efficacy Endocrinology and metabolism Female France Headache Health aspects Homocysteine Homocysteine - blood Homocystinuria Homocystinuria - drug therapy Human Genetics Human health and pathology Humans Inborn errors of metabolism Infant Inherited metabolic diseases Life Sciences Lung diseases Male Medical prognosis Medical research Medical screening Medicine Medicine & Public Health Metabolism Metabolites Methylenetetrahydrofolate reductase NMR Non-pharmacological intervention Nuclear magnetic resonance Patient monitoring equipment Patients Pediatrics Pharmaceutical sciences Pharmacology Pharmacology/Toxicology Plasma Population studies Rare diseases Registries Respiratory tract diseases Retrospective Studies Risk factors RoCH registry Safety Santé publique et épidémiologie Spain Studies Treatment Outcome Vitamin B12 Vitamin B6 Young Adult France Spain Europe Betaine anhydrous RoCH registry Efficacy Homocystinuria Safety
ISSN:	1750-1172, 1750-1172
Online Access:	Get full text
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