January 2018 updates

January 2018 Updates

I would like to begin by thanking all of you for your continued support and encouragement. I appreciate it immensely. I am so grateful to have such supportive colleagues all over the world.

Our drug-repurposing clinical trial of dantrolene sodium is ongoing. The trial began in January 2017. Twenty-two patients with Wolfram syndrome enrolled. Two patients decided to leave the study due to personal reasons and one patient could not start the study because of regulatory reasons. So nineteen patients started taking dantrolene sodium. As of today, fifteen of them have been taking dantrolene for more than 6 months. At this stage, our team has found few side effects and has identified tolerable dosage levels in adult and pediatric patients. We will find out more about the outcome of this study in the next several months.

I feel that something new and wonderful will happen to us this year. Thank you again for your continued support. I am hopeful and grateful.