• European Initiative Targets Diagnosis, Treatment of Rare Diseases
  • Cell Therapy CAP-1002 Improves Muscle, Lung Function in Boys with DMD, Interim Data Show
  • ‘Disabled’ vs. ‘Having a Disability’: Debating the Power of Words
  • Pfizer’s Gene Therapy Holds Promise in Treatment of DMD, Early Data Show
  • UberWAV Is the Next Best Thing for Accessible Transportation
  • Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows
  • $500K Gift to MDA Summer Camp Will Honor Robert Bennett Legacy
  • The Speak Foundation Speaks Loudly: A Prelude to September in Chicago
  • Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
  • Potential DMD Treatment, Pamrevlumab, Helps to Preserve Lung, Heart and Upper Limb Function in Youth, Early Trial Data Show
  • Here’s How I Explain My Experience with Duchenne to Others
  • MDA Awards Locana $550K to Advance Potential RNA-targeting Therapy for Myotonic Dystrophy