Mito in the News

“The approval of FORZINITY, the first treatment option for Barth syndromeA rare, genetic disorder of lipid metabolism that primarily affects males. and the first approved mitochondria-targeted therapeutic, is a pivotal victory for the Barth syndrome community and offers hope for […]

Company honored in its commitment to patients and families living with mitochondrialRelated to the mitochondria. disease; invites the community to walk with BPGbio colleagues and MitoAction BOSTON, MA — September 12, 2025 — BPGbio, […]

Earlier today, Saol Therapeutics shared the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the company’s application for dichloroacetateInvestigational drug for treatment of mitochondrial disease. A small inorganic molecule that increases the rate of oxidative phosphorylation, the main pathway by which the energy in food is converted (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency […]

FOR IMMEDIATE RELEASE [Boston, MA, April 1, 2024] – MitoAction, a leading nonprofit organization dedicated to improving the lives of individuals and families affected by mitochondrial disease, recently convened a […]

The Lurie Prize in Biomedical Sciences recognizes outstanding achievement by a promising young scientist (52 or younger) in biomedical research. It is worth $100,000, and awarded annually by the Foundation […]

https://cob.silverchair-cdn.com/cob/content_public/journal/bio/jam/10.1242_bio.060278/3/bio060278.pdf?Expires=1711827491&Signature=G4px-hX-CQRWqXepaWcuG0-Y-QYF06N9pma-LYMFv5E9CQq~vlJaOQI2uuSjvfqBwOSWi3OMmI79SiubfEXv94SN1ek0zC2F01FEYHL7El0mIQtZ-GTaXhO6IjvA-oOCPOcMpSjZomYdtICQ2E9FpDyRKP70OsoFX-c2qxbyQ6LnPrRgiFgfraB~UqADJy-WSqYrLgNvjSfnUXahRkSo45PxA9pSq8kAm8Pxjivc59PF27oDji~p2dufwrRQlCvYKzjcgaPRg43qMsmTh8DML0rdu1BZWnxueti5C-Rr6znyJPQQfRWDoQw1JVg28p9n15vtBkZjOIRdKg04gKVFSw__&Key-Pair-Id=APKAIE5G5CRDK6RD3PGA One of the great scientific discoveries of the 20th century is the extraordinary similarity among all living species at the molecular level, despite the extreme diversity of their appearance […]

https://www.frontiersin.org/articles/10.3389/fcell.2021.767407/full The application of DNA sequencing has led to tremendous progress in our understanding and diagnosis of mitochondrial disease, with mutationsgenetic variant, genetic change in hundreds of genes now established as causes. However, […]

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10183817/ https://pubmed.ncbi.nlm.nih.gov/37021792/ Every now and then there appears a truly novel proposal for alleviating the misery of mitochondrial disease. In this paper an international team presents such an idea. It’s […]

https://erictopol.substack.com/p/on-genome-editing-with-fyodor-urnov?utm_campaign=email-half-post&r=3we41&utm_source=substack&utm_medium=email#details Fyodor Urnov has been a pioneer and leader in genome editing, a term he invented, since that field began.  He is a U.C. Berkeley colleague of Jennifer Doudna, who […]

This paper is a brilliant advance in our scientific understanding of mitochondrial disease, though it does not lead immediately to a treatment.   https://www.pnas.org/doi/10.1073/pnas.2304884120 Leber hereditary optic neuropathy (LHON) is […]