• Sarepta Acquires Rights to Novel Gene Therapy Candidate to Treat Limb-Girdle MD
  • Let’s Prepare Ourselves for the Trials of a Lifetime
  • Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
  • Duchenne Teen Hits the Runway at Las Vegas Fashion Show
  • My Search for a Comfortable Bra as a Woman in a Wheelchair
  • Video Games Let Me Be Competitive Without the Need to Be Physical
  • #AANAM – FSHD Patients ‘Very Enthusiastic’ About Taking Part in Clinical Trials, Research Suggests
  • PPMD Launches Pilot Duchenne Newborn Screening Program in NY
  • #AANAM — New TTN Gene Variants Potentially Linked to Limb-girdle MD, Study Says
  • Find Your Communities and Gain a Sense of Belonging
  • #AANAM — Very Young Boys with DMD May Have Measurable Early Signs of Motor Skill Delay, Study Suggests
  • #AANAM —  Phase 1b/2 Trial Of RG6206 in Boys With DMD Supports Safety, Positive Muscle Effects