• EU Approval of Oral Cannabidiol as Add-On Therapy for Dravet Favored by CHMP
  • Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • Phase 1 Trial Planned for EPX-100, Potential Repurposed Therapy for Dravet, Epygenix Says
  • 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
  • Cannabidiol Oil Safely Lessens Seizures in Treatment-resistant Children, Early Trial Data Show
  • Encoded Earns $104M in Financing to Advance Potential Gene Therapy for Dravet
  • European Initiative Targets Diagnosis, Treatment of Rare Diseases
  • First Known Case of Double Somatic Mosaicism Reported in Girl with Dravet Syndrome
  • Zogenix Resubmitting New Drug Application to FDA for Fintepla Anti-Seizure Therapy
  • World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
  • Epygenix Submits Investigational New Drug Application for Dravet Therapy