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Research We Support

We focus our resources on supporting early-stage research that promises to slow the progression of Duchenne. Translating laboratory findings into cures is costly and risky, and we commit to supporting promising therapies through what is known in the drug development industry as the “valley of death.”

GalGT2

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.  Read more about GalGT2 in our public statement of November 2016.

AAV Re-Administration

Led by Dr. Keith Foster, this project seeks to address the challenges of adeno associated virus (AAV) delivered gene therapies, including immune response and systemic delivery strategies.

CRISPR-Cas9

A project supported by Parent Project Muscular Dystrophy and several Duchenne organizations, this gene editing therapy developed by Dr. Eric Olson shows promise to treat 80% of all Duchenne patients.  Read the press release here.

Follistatin

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.

Relaxin

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.

Quercetin

Determining the mechanisms whereby a quercetin enriched diet interrupts disease processes in DMD.

Parent Project Muscular Dystrophy

Duchenne Superhighway project to make clinical trial process faster and more efficient.

Halofuginone/HT-100

Anti-fibrotic/anti-inflammatory to keep muscle healthy and generate new muscle tissue.

Development of dystrophin independent therapy for DMD

Review of drugs to correct muscle degeneration using zebrafish model.

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