- Case Report
Large Benefit from Simple Things: High-Dose Vitamin A Improves RBP4-Related Retinal Dystrophy
- Vasily M. Smirnov,
- Baptiste Wilmet,
- Marco Nassisi,
- Christel Condroyer,
- Aline Antonio,
- Camille Andrieu,
- Céline Devisme,
- Serge Sancho,
- José-Alain Sahel and
- Christina Zeitz
- + 1 author
Inherited retinal diseases (IRD) are a group of heterogeneous disorders, most of which lead to blindness with limited therapeutic options. Pathogenic variants in RBP4, coding for a major blood carrier of retinol, retinol-binding protein 4, are respon...