“The approval of FORZINITY, the first treatment option for Barth syndrome and the first approved mitochondria-targeted therapeutic, is a pivotal victory for the Barth syndrome community and offers hope for […]
BPGbio Sponsors MitoAction Energy Walk at Boston’s Franklin Park Zoo on September 27th, 2025
Company honored in its commitment to patients and families living with mitochondrial disease; invites the community to walk with BPGbio colleagues and MitoAction BOSTON, MA — September 12, 2025 — BPGbio, […]
FDA denies approval for dichloroacetate (DCA), a treatment for Pyruvate Dehydrogenase Complex Deficiency (PDCD).
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 dichloroacetate (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency […]
MitoAction Advocates for Barth Syndrome Community in FDA Listening Session
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 […]
Mitochondrial Disease Researchers Win Major Award
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 […]
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