• Takhzyro Safely Prevents HAE Attacks Over Long Term, Extension Study Shows
  • Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
  • Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
  • Takhzyro, for HAE Attacks, Moves Closer to Public Funding Across Canada
  • Berotralstat Available to Eligible Patients in UK Through Early Access Program
  • Berotralstat Significantly Lowers HAE Attack Frequency, Phase 3 Trial Reports
  • NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
  • HAE Attack in Fetus Reported, Baby Responded to Mom’s Treatment
  • UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
  • Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
  • KalVista Plans Phase 2 Trial for KVD824, Will Develop Factor XIIa Preventive Treatment
  • Intellia’s NTLA-2002 Reduces Kallikrein Protein Levels for 11 Months in Preclinical Study