• Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA
  • Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
  • Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019
  • Tips to Alleviate Symptoms of Constipation
  • Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces
  • Defying the Odds of Living with Duchenne, Decade After Decade
  • PPMD Funds Gene Therapy Research Aimed at Improving Cardiac Function in DMD Patients
  • I Can’t Do Everything, and That’s OK
  • ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
  • DMD Increases Susceptibility to Rare, Aggressive Muscle Cancer, Mouse Study Suggests
  • FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
  • Popeyes Group Launches 15th Annual Campaign to Help MDA