Title:
What are the goals of the CFR?
The CFR has 4 main goals:
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Identify individuals with single large-scale mitochondrial deletion disorders, such as Pearson syndrome, Kearns-Sayre syndrome, and CPEO
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Gather past and future information on individuals with SLSMD through voluntary registration, self-reported survey questionnaires, patient-mediated medical record collection, and optional biospecimen sharing
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Make data collected through the CFR available to researchers and clinicians who are studying SLSMD disorders to answer questions regarding the disease, including its causes, potential treatments, and other topics
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Be the primary hub of clinical trial recruitment for individuals with SLSMD disorders