treat mito priorities

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TREAT MITO is dedicated to convening, organizing, and overseeing a unified research network for therapeutic development in mitochondrial diseases.   Some of the early priorities in accomplishing this include:  

•  Preclinical data review committee
  We have established a committee of mitochondrial disease experts who regularly meet to review preclinical data from industry interested in pursuing trials in mitochondrial medicine. The committee provides feedback on data and guides companies as to the best approach to develop a preclinical program targeting the primary mitochondrial disorders most suitable for their molecule/technology.
   

• Improving clinical trial readiness
  1. We have been assessing and analyzing clinical trial capacity for mitochondrial disease to help with site selection worldwide.
  2. We have organized, participated continue to engage in regulatory educational meetings to raise awareness about mitochondrial diseases and align goals between the community stakeholders and the FDA.
  3. We are developing clinical trial education for patients and investigators to improve trial participation and conduct.
  4. We are developing natural history studies leveraging patient and clinician registries.
  5. Supporting the development and validation of new outcome measures for primary mitochondrial diseases.
  (Modified fatigue Impact Scale, PROMIS mitochondrial disease Fatigue short form, Video assessment tools to measure functional impairment…) 

•  Identifying more mitochondrial disease patients by making genetic testing more accessible
  We have partnered with industry to help fund and support no-cost genetic testing for patients with suspected mitochondrial diseases. 

• Support with protocol development and regulatory agency interactions