September 9, 2026 New data presented in long-chain fatty acid oxidation disorders at INFORM 2026 support development of Company’s third mitochondrial-targeted candidate NEEDHAM, Mass., Sept. 9, 2026 /PRNewswire/ — Mighty Therapeutics (the “Company” or […]
First-Ever Mitochondrial Disease Advocacy Coalition Launched,Uniting 17 Groups Behind a Single Voice on Capitol Hill
September 3, 2026 New coalition brings together organizations from across the mitochondrial disease community to accelerate research funding, advance patient-centered legislation, and foster a responsive regulatory environment for new therapies. […]
Pretzel Therapeutics Advances PX578 into Phase 2 Study in Individuals Living with POLG-mediated Primary Mitochondrial Disease Following FDA Clearance of IND
August 25, 2026 PX578, a potentially disease-modifying, first-in-class investigational medicine cleared to start clinical trials in patients Initiation of Phase 2 Trial in POLG-PMD (POLG Disease) Planned for late 2026 […]
Mighty Therapeutics, Formerly Stealth BioTherapeutics, Announces New Name Honoring the Mitochondrial Disease Community
June 16, 2026 NEEDHAM, Mass., June 16, 2026 — Stealth BioTherapeutics Holdings Inc., a commercial-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for rare and […]
Khondrion Announces First Patient Dosed in Pivotal Phase 3 KHENERFIN Study of Sonlicromanol in Mitochondrial DNA 3243A>G Primary Mitochondrial Disease
April 16, 2026 NOTES TO EDITORSAbout KhondrionKhondrion is a late-stage clinical biopharmaceutical company focused on developing therapies forpatients with primary mitochondrial disease (PMD). Its lead asset, sonlicromanol, is a brain-penetrantredox […]
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