Role of Vitamin E in Gaucher Disease Patients

Summary

gaucher disease (GD) can be classified into three clinical types .type 1,the most common type ,is the chronic non neuronpathic form of the disease,which shows gighly variable signs and symptoms and variable course,with visceral,skeletal and hematologic involvement among others.the neurologocal involvement can be observed in types 2 and 3

Timeline

Start

2023-03-03

Primary completion

2023-09-05

Completion

2023-10-05

Publications

• Background Pacheco N, Uribe A. Enzymatic analysis of biomarkers for the monitoring of Gaucher patients in Colombia. Gene. 2013 May 25;521(1):129-35. doi: 10.1016/j.gene.2013.03.044. Epub 2013 Mar 21.
• Background Kartha RV, Terluk MR, Brown R, Travis A, Mishra UR, Rudser K, Lau H, Jarnes JR, Cloyd JC, Weinreb NJ. Patients with Gaucher disease display systemic oxidative stress dependent on therapy status. Mol Genet Metab Rep. 2020 Dec 9;25:100667. doi: 10.1016/j.ymgmr.2020.100667. eCollection 2020 Dec.
• Adly AAM, Ismail EAR, Ibrahim FA, Atef M, El Sayed KA, Aly NH. A 6-month randomized controlled trial for vitamin E supplementation in pediatric patients with Gaucher disease: Effect on oxidative stress, disease severity and hepatic complications. J Inherit Metab Dis. 2025 Jan;48(1):e12792. doi: 10.1002/jimd.12792. Epub 2024 Aug 25.

Drugs

Indications