Mitochondrial disease community registry (MDCR): Perspectives from patients and families, lessons learned from the data

Mrs. Sophia Zilber from Boston, USA will join the Targeting Mitchondria 2020 Congress and will give a presentation entitled "Mitochondrial disease community registry (MDCR): Perspectives from patients and families, lessons learned from the data".

Patient-populated registries are an important component of rare disease communities for many reasons, including their use as a tool for gathering opinions on specific topics. The Mitochondrial Disease Community Registry (MDCR) was launched in 2014 by United Mitochondrial Disease Foundation (UMDF) for this purpose as well as to identify and characterize mitochondrial disease patients from the patient perspective. This talk presents data collected over a four year period and provided by adult mitochondrial disease patients and caregivers of pediatric mitochondrial disease patients. Findings suggest the importance of clinician-patient communication, need for treatment and cure, the impact of the disease on the entire life of a person, and quality of life as top issues as described by patients, while also highlighting that patients are hopeful and optimistic.  Additionally, data quality checks showed that more clear and simple questions and shorter more-targeted surveys are needed in order to get accurate and meaningful data for research.

Targeting Mitochondria 2020 Congress
October 29-30, 2020
www.targeting-mitochondria.com