Betaine anhydrous in homocystinuria: results from the RoCH registry

Abstract 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 in...

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Auteurs principaux: Vassili Valayannopoulos, Manuel Schiff, Nathalie Guffon, Yann Nadjar, Angels García-Cazorla, Mercedes Martinez-Pardo Casanova, Aline Cano, Maria L. Couce, Jaime Dalmau, Luis Peña-Quintana, Vincent Rigalleau, Guy Touati, Luis Aldamiz-Echevarria, Pascal Cathebras, Didier Eyer, Dominique Brunet, Léna Damaj, Dries Dobbelaere, Claire Gay, Sylvie Hiéronimus, Virginie Levrat, François Maillot
Format: Artigo
Langue:Inglês
Publié: BMC 2019-03-01
Collection:Orphanet Journal of Rare Diseases
Sujets:
Betaine anhydrous
Efficacy
Homocystinuria
RoCH registry
Safety
Accès en ligne:http://link.springer.com/article/10.1186/s13023-019-1036-2
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