A Novel Therapeutic Strategy for Nephropathic Cystinosis

A way to attenuate kidney damage and enhance renal function in patients with chronic kidney disease (CKD).

Detailed article here.

Chronic Kidney Disease is a global health crisis affecting over 830 million people worldwide. Early stages of CKD are often asymptomatic, leading to late detection when the disease has progressed to irreversible kidney damage. Current treatments are insufficient in reversing kidney injury, particularly in conditions like nephropathic cystinosis—an inherited disorder leading to renal failure despite existing therapies. There is an urgent need for effective treatments that can halt or reverse the progression of CKD.

We are developing a novel formulation that enhances the bioavailability of protective compounds known for their renal benefits. This work includes creating the innovative formulation, conducting a pilot clinical study to assess its efficacy in reversing kidney damage, and measuring improvements in kidney function.

MTM students will engage in regulatory strategy planning, submission of IRB applications, patient recruitment and informed consent processes, clinical data collection, processing of the KIT assay in the lab, data analysis, business case development, financial modeling, and preparation of manuscripts and fundraising proposals.