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.