Real-World Study of Management and Outcomes of Patients with Lysosomal Acid Lipase Deficiency (LAL-D) in France
This descriptive study based on claims data confirmed the severity of LAL-D and the need to define the best management, considering the heterogeneity of the patients.
Institutions
- École Supérieure des Sciences Économiques et Commerciales (FR)
- Hôpital Necker-Enfants Malades (FR)
- Oracle (United States) (US)
- Inserm (FR)
- Sorbonne Université (FR)
- Alexion Pharmaceuticals (France) (FR)
- AstraZeneca (France) (FR)
- Pitié-Salpêtrière Hospital (FR)
- AstraZeneca (Switzerland) (CH)
- Fondation pour l’innovation en Cadiométabolisme et Nutrition (FR)
- Nutrition-Génétique et Exposition aux Risques Environnementaux (FR)
- Alexion Pharma (Switzerland) (CH)
- Université de Lorraine (FR)
Publication Details
- Journal
- Advances in Therapy
- Published
- 2026-03-24
- DOI
- https://doi.org/10.1007/s12325-026-03510-w
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00