Postauthorization safety study of betaine anhydrous.
E-HOD
betaine anhydrous
homocystinuria
orphan drug
postauthorization safety study
public private partnership
rare disease
Journal
Journal of inherited metabolic disease
ISSN: 1573-2665
Titre abrégé: J Inherit Metab Dis
Pays: United States
ID NLM: 7910918
Informations de publication
Date de publication:
07 2022
07 2022
Historique:
revised:
25
03
2022
received:
14
01
2022
accepted:
29
03
2022
pubmed:
1
4
2022
medline:
20
7
2022
entrez:
31
3
2022
Statut:
ppublish
Résumé
Patient registries for rare diseases enable systematic data collection and can also be used to facilitate postauthorization safety studies (PASS) for orphan drugs. This study evaluates the PASS for betaine anhydrous (Cystadane), conducted as public private partnership (PPP) between the European network and registry for homocystinurias and methylation defects and the marketing authorization holder (MAH). Data were prospectively collected, 2013-2016, in a noninterventional, international, multicenter, registry study. Putative adverse and severe adverse events were reported to the MAH's pharmacovigilance. In total, 130 individuals with vitamin B
Substances chimiques
Homocysteine
0LVT1QZ0BA
Betaine
3SCV180C9W
Methylenetetrahydrofolate Reductase (NADPH2)
EC 1.5.1.20
Cystathionine beta-Synthase
EC 4.2.1.22
Types de publication
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
719-733Informations de copyright
© 2022 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.
Références
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