Four Documentaries
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Joshua daily defied the odds and obstacles that were set before him. His passion, tenacity and love for life, powerfully impacted all who knew him. His life was a beacon of hope to those living around the globe. Joshua influenced many; from his fellow peers, to folks facing their own life struggles, to medical researchers studing neuromuscular disorders. Joshua’s life has impacted and will change the face of neuromuscular science as we know it, and The Joshua Frase Foundation will continue on its crusade in honor of Joshua and the Joshua’s of this world. Joshua's life will always be a reminder that #ChangeStartsWithOne.
2019 CNN HLN ~Something's Killing Me ~ Miracle Dog
To view our recent documentary ~ you can find this on On-Demand
In the summer 2012, Genthon in France, the University of Washington, Boston Children's Hospital and The Joshua Frase Foundation publicly released the collaborative effort of years of work on the advancements of Myotubular Myopathy. The synergistic work of the entities on the dog model, captured and engaged the investing of Venture Capital. This monumental advancement was the first of its kind ever in the neuromuscular space in a large animal model. Their work in gene therapy showed proof of concept with complete correction in a naturally occuring muscle disorder in the canine animal model forging the path for clinical trials.
New step in the treatment of myotubular myopathy by gene therapy
Nouvelle étape dans le traitement de la myopathie myotubulaire par thérapie génique
Gene Therapy Rescues a Fatal Neuromuscular Disease in Dogs
Our Scientist, Martin Childers of the University of Washington, studying myotubular myopathy, a devastating disorder, say a new therapy appears to rescue mice and naturally occuring dogs from the disease. The findings demonstrate the feasibility of future clinical trials of gene therapy.