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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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Hoofdauteurs: Geetanjoli Banerjee, Sue Perera, Fan Mu, Erin Cook, Mu Cheng, Adina Zhang, Jessie Jie Lan, Lin Zou, Vanja Sikirica
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: BMC 2025-07-01
Reeks:Orphanet Journal of Rare Diseases
Onderwerpen:
Propionic acidemia
Metabolic decompensation event
Propionyl coenzyme A carboxylase
Clinical outcomes
Age stratification
Administrative claims data
Online toegang:https://doi.org/10.1186/s13023-025-03901-2
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