• Muscular Dystrophy Association Awards 13 Research Grants Totaling $3.5 Million
  • Molecules Identified That Target Gene Known to Cause FSHD in Study
  • Exonics Secures $40M in Financing for Gene Editing Program to Treat Duchenne
  • Protein Utrophin Reduces Mitochondria Damage in Duchenne MD, Study Shows
  • FDA Paves Way for Clinical Trials of Sarepta’s Duchenne MD Therapy SRP-5051
  • Wave Begins Phase 1 Trial of Therapy That Addresses Exon Skipping in Duchenne MD
  • FDA Clears IND Application for Micro-dystrophin Gene Therapy Program to Treat Duchenne
  • Everest to End Duchenne Raises $100K to Fund CRISPR Gene-Editing Research
  • Chronic Illness and Offers of Prayers
  • FDA Clears IND Application for GALGT2 Gene Therapy for Duchenne Muscular Dystrophy
  • Bionano Genomics Platform May Shorten Time to Duchenne Diagnosis, Researchers Suggest
  • Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’