• Nonprofits RARE Courage, All Wheels Up Partner on Survey Seeking to Improve Air Travel for Wheelchair Users
  • FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
  • With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
  • Khondrion Doses First Patient in Phase 2b Trial Testing Sonlicromanol for Mitochondrial Diseases
  • Entrada Developing Enzyme Replacement Therapy ENTR-501 to Treat MNGIE Disease
  • NeuroVive Soon to Recruit Patients With Primary Mitochondrial Disease to Test its Oral Treatment Candidate
  • Elamipretide Failed to Meet Promise of Earlier Trial Results for Primary Mitochondrial Myopathy, Data Show
  • New Fluorescent Marker Allows Detailed Study of Mitochondria in Live Cells, Study Reports
  • LHON Natural History Study Highlights Improvements in Visual Acuity From Lumevoq Treatment
  • Gene Therapy Takes Center Stage at 2019 NORD Summit
  • New Mouse Model May Advance Understanding of Mitochondria Protein Production and Dysfunction, Researchers Say
  • HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases