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EPISODE · Dec 17, 2021 · 41 MIN

Mitochondrial Disease Special Issue: Novel Therapies

from JIMD Podcasts · host Journal of Inherited Metabolic Disease

In March 2021 our special issue look at all aspects of Mitochondrial Disease. In this special episode Professor Shamima Rahman guest hosts as we welcome David Dimmock, Mike Lawlor, Guilhian Leipnitz and Marc Patterson to discuss their papers from that issue, looking at novel therapies in mitochondrial disease. For those skipping: DGUOK (from 3min 30sec), SO deficiency (from 19min) and Friedreich's Ataxia (29min 10sec). The nucleotide prodrug CERC-913 improves mtDNA content in primary hepatocytes from DGUOK-deficient rats Vanden Avond, et al https://doi.org/10.1002/jimd.12354 The mitochondrial-targeted reactive species scavenger JP4-039 prevents sulfite-induced alterations in antioxidant defenses, energy transfer, and cell death signaling in striatum of rats Glänzel, et al https://doi.org/10.1002/jimd.12310 Safety and efficacy of (+)-epicatechin in subjects with Friedreich's ataxia: A phase II, open-label, prospective study Qureshi, et al https://doi.org/10.1002/jimd.12285

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