Clinical burden of propionic acidemia in the United States: a claims-based study by age stratum

Abstract Background Patients with propionic acidemia (PA) may face recurrent metabolic decompensation events (MDEs) and multisystemic complications. This study compared characteristics and clinical outcomes of patients with PA and matched non-PA controls by age stratum. Methods Patients with PA from...

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Autors principals:	Geetanjoli Banerjee, Sue Perera, Fan Mu, Erin Cook, Mu Cheng, Adina Zhang, Jessie Jie Lan, Lin Zou, Vanja Sikirica
Format:	Artigo
Idioma:	Inglês
Publicat:	BMC 2025-07-01
Col·lecció:	Orphanet Journal of Rare Diseases
Matèries:	Propionic acidemia Metabolic decompensation event Propionyl coenzyme A carboxylase Clinical outcomes Age stratification Administrative claims data
Accés en línia:	https://doi.org/10.1186/s13023-025-03901-2
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