• Taylor’s Tale Co-founder Races to ‘Change the World’ for Batten Disease Community
  • Extracellular Vesicles May Enhance Delivery of TPP1 to the Brain for Replacement Therapy in CLN2, Early Study Suggests
  • ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
  • At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
  • Can Patients and Families Really Drive Therapy Development?
  • Amicus CEO John Crowley Outlines Progress in Batten, Pompe, Fabry Therapies
  • Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • Neurogene Opens Natural History Study of CLN7 and CLN5 Disease at UT Southwestern
  • Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
  • Amicus, Paragon to Collaborate on Gene Therapies for Batten, Other Diseases
  • European Initiative Targets Diagnosis, Treatment of Rare Diseases