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April 2019


On March the 29th a patient-focused drug development meeting was organised with the Food and Drug Administration in the USA. The organisors being the United Mitochondrial Disease Foundation, MitoAction and the Muscular Dystrophy Association.

The main purpose of the meeting was to establish good communication between FDA and mitochondrial patients, so that pharmaceutical regulators such as the FDA learn about mitochondrial disease from a patient’s perspective, about the disease burden and ideal treatments. If there is good understanding of this, it may smoothen the way for new therapies and drugs to be authorised for use by patients. This is not only valuable for patients in the USA but for patients in the whole world.


Following this we would like to ask as many patients with mitochondrial disease as possible and/or their caregivers to complete a survey which was set up. The data coming out of this survey will be used to create a VOICE OF THE PATIENT report which will be freely available for all interested parties. Importantly, pharmaceutical regulators such as the FDA (and possibly others such as the EMA) will utilize the report as reference material to have better insight and understanding of the wants and needs of mitochondrial patients, so that this can help them with those difficult therapeutic approval decisions.


The Survey

If you are 1) an adult patient or a caregiver for someone with muscle-based symptoms (myopathies) or 2) a caregiver of a pediatric patient with brain-based symptoms (neurologic), then please complete the


2019 Patient/Caregiver Voice of the Patient Survey


This is a worldwide survey, not restricted to the USA,  and will be open to responses up to the 30th of April, 2019.

You can remain anonymous if you prefer.

Questions on the survey can be asked through This email address is being protected from spambots. You need JavaScript enabled to view it.


Please share this request with your own network.


More information:







© 2019 Int. Mito Patients