• Rare Disease Film Festival Highlights Patient and Researcher Unity
  • HMBS Mutations Linked to Acute Intermittent Porphyria May Vary According to Ethnicity, Study Suggests
  • Recurrent Attacks in AHP Patients Serious and Lead to Chronic Symptoms, Study Based on Natural History Finds
  • FDA Approves Scenesse to Increase Pain-Free Light Exposure for Adults with EPP
  • Targeting ABCG2 Protein Seen to Prevent Skin Blistering, Liver Damage in Mouse Model of EPP
  • NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
  • 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