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 […]
Navigating the FDA’s Complete Response Letter and identifying a path forwardfor SL1009 (DCA).
March 31, 2026 Dear Members of the PDCD Community, We are deeply grateful for your support and advocacy throughout this process, particularly aswe have worked to navigate the FDA’s Complete […]
FDA Approval of KYGEVVI Marks a Defining Moment for Mitochondrial Disease Community and Families Affected by TK2d
FOR IMMEDIATE RELEASE Contact:Emily GrandahlMarketing Coordinator, MitoActionemily@mitoaction.org(888) 648-6228www.mitoaction.org FDA Approval of KYGEVVI Marks a Defining Moment for Mitochondrial Disease Community and Families Affected by TK2d Boston, MA — November 3, […]
Tisento Therapeutics Announces Publication of MELAS Interview Study, Incorporating Patients’ Perspectives to Elucidate Symptoms and Impacts of Rare Mitochondrial Disease
Originally Published by Tisento Therapeutics on November 3, 2025. Findings Demonstrate the Importance of Addressing Both Fatigue and Cognitive Symptoms of MELAS CAMBRIDGE, Mass., November 3, 2025 – Tisento Therapeutics […]
Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome
“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 […]
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