• FDA Explands Patisiran’s Orphan Drug Status to Another Form of Familial Amyloidosis
  • Cognitive Impairment Common Among Older, Untreated FAP Patients
  • MMP-14 Levels May Serve as Biomarker and Therapeutic Target in FAP Patients
  • Alnylam Starts Rolling Submission of Application Asking FDA to Approve Patisiran
  • Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US
  • Vyndaqel Can Slow Nerve-cell Damage in FAP, Regardless of Gene Mutation, Analysis Finds
  • EMA Regulatory Committee Grants Accelerated Assessment to Patisiran to Treat FAP
  • Inotersen, Potential FAP Treatment, Under Review for Approval in Europe
  • Alnylam Set to Seek Regulatory Approval for Patisiran With Robust Findings in Phase 3 FAP Trial
  • Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’
  • FAP Patients Have a Moderate Chance of Having Unaffected Children, Study Suggests
  • Psychological, Psychiatric Support Can Be Beneficial for TTR-FAP Patients, Study Suggests