Drs. Sebahattin Cirak and Yetrib Hathout

Children’s National Medical Center (Washington, DC)


Project Summary:

Surrogate biomarkers are needed to predict clinical and biochemical outcomes to guide clinical trials and monitor treatment. This project will analyze plasma samples from LGMD2I patients and compare them with samples from patients with other sub-types of dystroglycanopathies and with healthy control subjects. The goal is to identify proteins and microRNAs markers present in these samples that give insights into the different stages of progression of these diseases and that can be used as biomarkers in future clinical trial. These surrogate biomarkers should lead to cost-effective and patient friendly clinical trials and treatment.

This grant is jointly-funded by the LGMD2I Research Fund, the Samantha Brazzo Foundation and Cure CMD.