• Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28
  • Antibiotic Preventive Treatment Rates for IPD Very Low Among Sickle Cell Children, Study Says
  • Imara Begins Phase 2 Sickle Cell Disease Trial Testing IMR-687 in Adults
  • Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress
  • Hypersensitivity to Allergens May Increase Risk of Acute Chest Syndrome in Sickle Cell Anemia Children
  • Two Types of CRISPR Gene Editing Could Be Useful in Treating Sickle Cell Anemia, Editas Reports
  • Hydroxyurea Treatment in Men with SCA Leads to Drop in Total Sperm Count, Study Shows
  • Medicaid to Cover Endari for Sickle Cell Disease in the U.S.
  • NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases
  • How a Man and Woman, Both with a Sickle Cell Anemia Mutation, Had a Healthy Child
  • Entrepreneur to Design New Voice-recognition Mobile App for Monitoring Blood Disease
  • Endari Now Available by Prescription in US for Sickle Cell Disease Treatment