What We Are Funding

First Key: Facilitate Awareness

Second Key: Fund Research For Improved Quality Of Care

  • Support The Pediatric Neuromuscular Clinic at Massachusetts General Hospital for Children
    • Provide quality medical treatment while practicing bench to bedside research
    • Donate items to entertain patients during long appointments including ipads, toys and books
  • Fund research to improve physical complications of DMD
    • Lung Function Study, Sleep Study, Stander Wheelchair Study, Bone Density Study
  • Fund the Massachusetts General Hospital for Children Pediatric Neuromuscular Clinic Registry
  • Fund Research for Improved Pharmacologic Treatments

Third Key: Fund Advancing Research For A Cure

  • Member of and contributor to The Duchenne Alliance
    • A group of independent Duchenne organizations committed to a cure that can advance research faster than anytime before
  • Help fund Follistatin gene therapy trial 
    • A growth-stimulating protein that improves muscle strength. Currently enrolling DMD patients for the trial. 
  • Help fund the manufacturing of GALGT2 that is introduced into the body using a viral vector in a surrogate gene therapy trial
    • Leads to expression of GALNAC transferase across the entire muscle membrane, as well as upregulation of utrophin across the entire muscle fiber. Currently enrolling patients.
  • Help fund reseach of Nutraceutical Quercetin
    • A treatment to improve respiratory and cardiac function. Initial findings promising.  Will continue further pre-clinical research.
  • Help fund the research for Relaxin
    • A natural hormone that hopes to enhance regeneration, increase strength, and reverse fibrosis in all types of muscles.  Preclinical trials promising. Will move to patient trials.