• Data Support Long-term Efficacy of Berotralstat in HAE, BioCryst Says
  • Coalition Will Address Racial Disparities in Rare Disease Communities
  • Patent Allowance Extends BioCryst’s Protection of Berotralstat for HAE
  • Prefilled Syringe for At-home Use of Takhzyro Favored for Approval in EU
  • COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
  • Intellia Advancing NTLA-2002 as Gene-Editing Therapy Candidate for HAE
  • Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
  • Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
  • Costs Unsustainable for New Angioedema Preventive Therapies in US, Real-world Data Show
  • Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
  • European Commission Approves Ruconest for Acute Swelling in Children with HAE