• TB Antibiotic Shows Promise in Mice with Erythropoietic Protoporphyria, but Researchers say More Human Studies are Needed
  • Givosiran Continues to Show Promise in Treating AHP, Trial Data Shows
  • Glycyrrhizin May Be Effective in Patients with Alcohol-linked PCT, Case Report Says
  • ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
  • PCT Linked to Higher Risks of Gastrointestinal Disease, Cancer, and Premature Death, Study Suggests
  • At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
  • Alnylam  Partners with Ironwood to Make Doctors More Aware of Givosiran’s Potential
  • Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • FDA Grants Priority Review to Givosiran for Treating Acute Hepatic Porphyria
  • New Mutation in FECH Gene Linked to EPP, Study Shows
  • 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