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  • FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development
  • CHMP Opposes Approval of Exondys 51 to Treat DMD in Europe
  • Canada Trails US in Patient Access to Rare Disease Therapies, CORD President Says
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  • Dating with a Disability: Part 1
  • EU’s CHMP Recommends Translarna to Treat Younger DMD Children After FDA Rejection
  • US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases