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November 2024 Newsletter: FDA News, Rare Revolution Magazine Feature, Therapeutic Pipeline Updates, and more
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Ask the Mito Doc – October 2024; Q&A
Ask the Mito Doc - October 2024 Q&A All answers today are based on personal experience of the participants. As always, please consult your personal physician prior to taking any action.Connecting the Dots Between Mito and Rare DiseasesConnecting the Dots...
Advocacy Update – Oct 2024: FDA AdComm Brings Good News, Plus DC End of Year Business
FDA Advisory Committee Deems Potential Barth Syndrome Drug "Effective" Earlier this month, an FDA Advisory Committee voted 10-6 supporting approval of Stealth BioTherapeutics’ elamipretide for treating Barth syndrome, a rare mitochondrial disease that occurs mostly in...