Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.

ABSTRACT: Current management guidelines for urea cycle disorders (UCDs) offer clear strategies, incorporating both authorized and non-authorized medicinal products (including intravenous formulations and products regulated as food). These varying product categories are subject to specific accessibility challenges related to availability, reimbursement, and pricing. The aim of this study is to identify potential obstacles to optimal UCD treatment implementation in European clinical practice. A survey aimed at metabolic healthcare professionals (HCPs) managing patients with UCDs in Europe was disseminated through the European Reference Network for Hereditary Metabolic Disorders and the European registry and network for intoxication type metabolic diseases. Forty-eight survey responses were collected f

SUBMITTER: Stolwijk NN 

PROVIDER: S-EPMC11670152 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature