There are two new MCADD trials that have started in recent months. One explores the use Dojolvi (triheptanoin) and the second the use of phenylbutyrate with MCADD patients. Join MitoAction […]
2024 Mito Town Meeting
The annual mito town meeting is our way of kicking off the new year by sharing all that we have in store for the next 12 months! We will hear from organizations […]
Expert Series: Ultra-Rare Mitochondrial Diseases: Development Challenges and Opportunities
Join CEO of Stealth Biotherapeutics Reenie McCarthy, and special guest Shelley Bowen, Director of Family Services and Advocacy for the Barth Syndrome Foundation, as they discuss the health equity challenges […]
Expert Series: Ceramides: The Unmasked Drivers of VLCADD-Induced Heart Failure
Despite advances in newborn screening and treatment of fatty acid oxidation disorders, patients with very-long-chain acyl-CoA dehydrogenase deficiency (VLCADD) continue to suffer from heart failure. Marie Norris will discuss her […]
Expert Series: LCHADD Retinopathy Update: Moving Toward a Treatment
Having a better understanding of what things are associated with LCHADD retinopathy can provide insight towards developing treatment. During this presentation, Dr. Gillingham will provide an update on the current […]
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