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Clinical Trials


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...


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...


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...


Moving Mito Medicine: Reneo Strides Study

On Friday, June 4 at 12:00pm EST, we will be joined by Dr. Madhu Davies from Reneo Pharmaceuticals for our June Mito Expert Series presentation, titled Moving Mito Medicine: Reneo Strides Study. About...


Clinical Trials: Why you should care and why you should participate. How do we know what we know?

Join MitoAction and Dr. Jerry Vockley from University of Pittsburgh Children’s Hospital on Friday, May 14, 2021 for our monthly expert series presentation!     About the Speaker Gerard Vockley, MD,...


Patients as Partners in Drug Development

Join MitoAction and Dr. Madhu Davies from Reneo Pharmaceuticals for our February Monthly Mito Expert Series titled “Patients as Partners in Drug Development.” About the Speaker: Dr. Madhu Davies, MB, ChB,...


PTC743 (Vatiquinone) for the Treatment of Mitochondrial Disease With Associated Epilepsy

Join MitoAction and Matthew Klein and Francesco Bibbiani from PTC Therapeutics for our November Mito Expert Series presentation titled, “PTC-743 (Vatiquinone) for the Treatment of Mitochondrial Disease With Associated Refractory...


Ultragenyx Q&A – A Conversation with Ultragenyx’s CMO and Global Metabolic Dietitian

A Conversation with Ultragenyx’s Chief Medical Officer, Camille Bedrosian to discuss Insights into Future LC-FAOD Research.  Also hear from Ultragenyx’s Global Metabolic Dietitian, Bridget Reineking on How to Use...



Stealth BioTherapeutics MMPOWER Update

This summer, Stealth BioTherapeutics (Stealth BT) shared preliminary positive data from the MMPOWER study. The study purpose was to evaluate safety and efficacy of elamipretide (previously known as Bendavia)...