• Funding Supports Pharnext’s Phase 3 Trial of PXT3003
  • Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
  • Rare Disease Day at NIH, Set for March 1, Growing Year by Year
  • Rare Disease Day Reminds Me of the Importance of Community
  • More Research Needed into Effects of Exercise on CMT Patients, Review Says
  • Gene Therapy Seen to Rescue Motor, Nerve Defects in CMTX1 Mouse Model
  • CMT Research Foundation Funds Work Into Way of Delivering Gene Therapy
  • Mutations in LRSAM1 Gene Tied to Rare CMT Subtype
  • Pandemic Won’t Stop Rare Disease Day on Feb. 28
  • Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
  • Pivotal CMT1A Trial to Begin Patient Enrollment Soon
  • NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns