Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Services In Belgium
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by:Genome Biologics based inFrankfurt am Main, GERMANY
Kearns-Sayre Syndrome (KSS) is a mitochondrial disease, with Rare Disease designation (7,400 – 22,000 patients in Europe), caused primarily by large-scale mitochondrial DNA deletions. Cardiac dysfunction is the principal clinical manifestation and primary cause of death. There are no current treatment options available that go beyond addressing some of the symptoms associated with KSS thus ...