Societal costs and quality of life associated with arginase 1 deficiency in a European setting – a multinational, cross-sectional survey-Reference-Cited by-同舟云学术

Societal costs and quality of life associated with arginase 1 deficiency in a European setting – a multinational, cross-sectional survey

Published:2024-09-07 Issue:1 Volume:27 Page:1146-1156
ISSN:1369-6998
Container-title:Journal of Medical Economics
language:en
Short-container-title:Journal of Medical Economics

Author:

Olofsson Sara¹^ORCID,Löfvendahl Sofia¹^ORCID,Widén Julia²^ORCID,Rudebeck Mattias²^ORCID,Lindgren Peter¹³^ORCID,Stepien Karolina M.⁴^ORCID,Arnoux Jean-Baptiste⁵,Luz Couce Pico Maria⁶^ORCID,Leão Teles Elisa⁷,Jacobson Lena²^ORCID

Affiliation:

1. The Swedish Institute for Health Economics, IHE, Lund, Sweden

2. Immedica Pharma AB, Stockholm, Sweden

3. Karolinska Institutet, Stockholm, Sweden

4. Salford Royal Organization, Northern Care Alliance NHS Foundation Trust, Salford, United Kingdom

5. Necker Hospital, Paris, France

6. Hospital Clinico Universitario de Santiago de Compostela, IDIS, MetabERN, Santiago de Compostela, Spain

7. Centro Hospitalar Universitário de São João, MetabERN, Porto, Portugal

Funder

Immedica Pharma AB

Publisher

Informa UK Limited

Link

https://www.tandfonline.com/doi/pdf/10.1080/13696998.2024.2400856

Reference39 articles.

1. The incidence of urea cycle disorders

2. Arginase 1 Deficiency: using genetic databases as a tool to establish global prevalence

3. Diaz G Longo N Bubb G et al. Delays in diagnosis are associated with poor clinical outcomes in patients with Arginase 1 deficiency. Poster presented at the 2019 European Paediatric Neurology Society (EPNS) Congress 17–21 September 2019 Athens Greece; 2019.

4. Health care resource utilization in the management of patients with Arginase 1 Deficiency in the US: a retrospective, observational, claims database study

5. Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports