This presentation will discuss lactic acidosis and focus on pyruvate dehydrogenase as a case study to explore how compassionate use and clinical trials can be used in patients with mitochondrial […]
Stealth BioTherapeutics & NuPOWER Trial Update
Stealth’s lead investigational compound elamipretide targets the inner mitochondrial membrane, binds to cardiolipin and has been shown to improve mitochondrial function. It is being studied in rare diseases linked to […]
LC-Fatty Acid Oxidation Disorders: Recent Updates on the REN001 Study
There was much discussion about current research in FAODs at the 2022 International Metabolic conference. Reneo Pharmaceuticals recently released positive results from the REN001 Phase1b LC-FAOD Study. Dr. Alex Dorenbaum, […]
MELAS Spectrum Disorders and Sonlicromanol: Progress Update
Join MitoAction and Dr. Jan Smeitink, CEO of Khondrion for our our March Expert Series presentation. MELAS spectrum (m.3243A>G) disorders belong to the most frequently encountered group of primary mitochondrial […]
The Mito Disease Drug Development Journey
Join Dr. Matt Klein from PTC Therapeutics , MitoAction and Cure Mito for an exclusive webinar discussing the history of clinical research in Leigh Syndrome, the evolution of PTC-743 and […]
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