Improve Quality Of Care & Life For Those Living With DMD
A treatment for heart failures and a potential skeletal muscle enhancer. http://phrixuspharmaceuticals.com/
Follistatin gene therapy trial
Phase I/IIa trial in boys with Duchenne following a trial in Becker muscular dystrophy patients. Follistatin inhibits the myostatin pathway, which has shown to cause significant enlargement of muscle mass and increased muscle strength.
A gene editing therapy shows promise to treat 80% of all DMD patients.
Determining the mechanisms whereby a quercetin enriched diet interrupts disease processes in DMD.
Fund Advancing Research For A Cure
A surrogate gene therapy for DMD that uses a viral gene transfer vector to deliver the GALGT2 gene. In animal models, delivery of GALGT2 results in a change to glycosylation across the entire muscle membrane, with upregulation of utrophin and other proteins that stabilize the muscle membrane, resulting in correction of muscle pathology and force deficits. This is a particularly promising approach because it is a potential therapy for any boy with DMD, regardless of mutation.
Relaxin is a natural hormone, which has a number of biological effects that may be of benefit in Duchenne Muscular Dystrophy (DMD), including its ability to regenerate muscle, reduce scarring and improve cardiac function.