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6 | InTouch AUTUMN 2023 The Neurogenetic Research Group, led by Associate Professor Richard Roxburgh and managed by Miriam Rodrigues is growing and we look forward to introducing our new staff and collaborators in the next issue. We have an exciting year underway already with plans to share some of our research with the wider community at the CBR Neurogenetics Research Seminar on Friday 10th March at the University of Auckland. All welcome. Participants with mitochondrial myopathy in the STRIDE clinical trial are now all transitioning over to STRIDE AHEAD, which is an open- label study designed to evaluate the long-term safety and tolerability of REN001. Reneo Pharmaceuticals has developed REN001, which they say is a potent and selective agonist of the peroxisome proliferator-activated receptor delta (PPARδ) that has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation and promote formation of new mitochondria. We are currently recruiting participants with mitochondrial myopathy caused by genetic alterations in the nuclear (not mitochondrial) genome. The trial, sponsored by Stealth Biopharmaceuticals, seeks to demonstrate the effectiveness of elamipretide in mitochondrial conditions. The criteria for the trial RESEARCH NEWS Miriam Rodrigues shares exciting updates at The Neurogenetic Research Group. Striding ahead with plans for 2023 includes having chronic progressive external ophthalmoplegia (CPEO), which is weakness of the muscles around the eyes, as well as muscle weakness or exercise intolerance. Please email neurogenetics@adhb. govt.nz if you’re interested in learning more about this clinical trial. Clinical trials occur more successfully on a solid background of knowledge and understanding about the condition, which is why we’re passionate about natural history studies. Natural history studies don’t trial a particular treatment but record observations about the condition. Many participants Above: REN001 is an investigational clinical-stage compound known to control a number of genes involved in the mitochondrial activity Recruitment for all our clinical trials and natural history studies takes place through Pūnaha Io the New Zealand Neurogenetic Registry & Biobank. If you would like to learn more about any of our clinical trials or other studies please email: neurogenetics@adhb.govt.nz Clinical trials occur more successfully on a solid background of knowledge and understanding about the condition, which is why we’re passionate about natural history studies. in natural history studies enjoy the experience and benefit from increased medical care, attention, and appropriate referrals. At the CBR Neurogenetic Clinic we are enrolling adults with Friedreich Ataxia , spinal muscular atrophy , and myotonic dystrophy in natural history studies. Miriam Rodrigues