• #AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD
  • #AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows
  • Noticing the Blessings in Everyday Life
  • UT Researcher, with Cure Duchenne Support, Launches Company to Treat DMD Using CRISPR/Cas9 Technology
  • NIH Agency Pioneers Collaborative Research into Rare Diseases
  • Pfizer Launches Phase 1b Clinical Trial for Mini-Dystrophin Gene Therapy to Treat DMD
  • Breaking the Stigma of Asking for Help
  • Genetic Compensation Mechanism Could Aid in Treatment of Muscle Disorders, Study Suggests
  • PPMD Names PerkinElmer to Conduct Genetic Tests for Its Decode Duchenne Program
  • Santhera’s Omigapil Found Safe and Well-Tolerated in Young CMD Patients in Phase 1 Trial
  • Connecticut Neurologist Upbeat on New Therapies for DMD, Other Disorders
  • New Gene Editing Strategy Leads to Much Longer Protein Production