Taurine as a therapy for Duchenne muscular dystrophy (DMD), and Becker muscular dystrophy (BMD)

Peter Arthur
University of Western Australia

Tuesday 24th Ocotber 2017

This is a proposal from Dr. Peter Arthur and colleagues at the University of Western Australia to develop Taurine as a potential therapy for the treatment of Duchenne and Becker muscular dystrophy.  The proposal seeks to establish blood and urine biomarkers that respond to Taurine treatment and use these biomarkers in a dose-escalation clinical study to identify an optimal taurine dose. The primary goal is to demonstrate improvements in muscle function and echocardiography in a cohort of boys with DMD in a longer-term clinical trial.

TACT welcomed the opportunity to review the proposal at this stage where discussion impacts not only the design of the clinical study, but also considered the potential for additional studies to aid biomarker selection. The committee felt that the program demonstrated a plausible basis for expecting a beneficial effect in DMD patients and generally supported the further investigation of taurine in the clinic. The report makes recommendations on further pre-clinical studies that include an examination of potential biomarker candidates in animal models as part of the biomarker selection process. TACT also makes recommendations on the consideration of the wide availability of Taurine and its presence in the background diet, manufacturing controls, IP protection and regulatory advice.
To request a full copy of the report please contact the applicant: Peter Arthur

20 Dec 2017