• mTOR Inhibitors May Benefit Some Children With Mitochondrial Diseases, Case Series Suggests
  • Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • Protein’s Inability to Bind, Stabilize Mitochondrial Membrane Drives Optic Atrophy, Study Suggests
  • Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
  • Trial of Cholesterol Medication in Barth Syndrome Patients Opens in UK
  • European Initiative Targets Diagnosis, Treatment of Rare Diseases
  • GenSight Completes Enrollment Early for Phase 3 Trial of GS010 Gene Therapy
  • New Lab Model Could Shed Light on Mitochondrial Epilepsy and Therapies
  • World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
  • Phase 1/2 Trial of Mitochondrial Cell Therapy for Pearson Syndrome Starts Dosing
  • NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala