Sonlicromanol in Primary Mitochondrial Disease MELAS Spectrum Disorders

Dr Jan SmeitinkDr. Jan Smeitink, from Khondrion B.V., Netherlands, will join us this year to present his most recent findings on "Sonlicromanol in Primary Mitochondrial Disease MELAS Spectrum Disorders" .

MELAS spectrum disorders caused by the 3243A>G mutation in the MT-TL1 gene lead to hampered oxidative phosphorylation with, as immediate cellular consequences, both reductive and oxidative stress.The clinical spectrum is heterogeneous with classical mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) and maternally inherited diabetes mellitus and deafness (MIDD) as most frequently encountered phenotypes. Sonlicromanol modulates key metabolic and inflammatory pathways in the pathogenesis of MELAS-SD models.

Here, Dr. Smeitnik reports the results of preclinical and phase 1 and 2 clinical studies which all together will guide the design of the upcoming pivotal phase 3 KHENERFIN trial.

Targeting Mitochondria 2022 Congress
October 26-28, 2022 - Berlin, Germany 


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