Sun, Feb 19, 2017
Gene therapy treats muscle-wasting disease in dogs
Transfer of the MTM1 gene into the weakened muscle tissue of dogs affected by myotubular myopathy restores muscle strength. The disease arises from a genetic mutation that disrupts the production of a protein needed for muscle function, Replacing the gene appears to be corrective. Click here to read the press release.
One little boy, who was granted just 15 years on this earth, touched a lot of people for the good.
Joshua Frase was born with myotubular myopathy (MTM), a rare genetic mutation of the MTM1 gene that makes it difficult for, primarily males, to crawl, walk and breathe on their own. His parents, Paul Frase, a former NFL defensive lineman for the Jacksonville Jaguars, and wife, Alison Rockett Frase, founded the Joshua Frase Foundation when Joshua was born with the disease.
Typically 70 percent of children born with MTM never celebrate their second birthday.
“My son Joshua’s Journey was a struggle between life and death,” said Rockett Frase. “His perseverance and character gave us guarded hope that he would live and not die. He inspired us to raise awareness and forge a path to find a cure for him and his peers.”
Dear Faithful Partners,
It is hard to believe it has been 21 years since Joshua was born and that this Christmas Eve will mark 6 years since his passing. 20 years ago the Joshua Frase Foundation (JFF) asked donors to invest in a guarded dream of one day finding a cure for myotubular myopathies. In those early years, our dreams as parents were laden with a distant hope, and a plea. Today, it is a proven science, moving closer to a cure. This dream has become a reality, only because of your help and investment.
We have learned that a message of hope can infiltrate the impossibilities of modern science.We have learned that new breakthroughs only come from pushing the boundaries of science beyond perceived limitations. We have learned that among the tireless efforts of many, science has no limits, and answers come to those who seek. In the case of myotubular myopathy research, the answers are coming in more than one form, and more than one scientific technology.
When we first viewed the video sent to us by one of our researchers showing the crippled mouse flourishing after one injection of gene replacement therapy, I was brought to tears, thinking: could this be a potential treatment for our son? Looking back on what has transpired in less than 10 years, I am in awe and I am humbled by those of you who invested in this guarded dream. You have given without hesitation, and you’ve trusted us to search out the most promising science, and we have been faithful with your investment dollars. This ultra-orphan disorder was brought to a pre-clinical stage of research with less than 10 million dollars. Within the scientific community, this is no small feat.
On the horizon…
JFF continues to live in the trenches of patient advocacy. I am reminded daily that this disorder knows no bounds. It is not a respecter of persons. It touches every continent and every walk of life.
Despite this reality, our goal of funding efforts to find therapies and a cure remainssteadfast. One of our long standing donors, Amy Groshell, says this of JFF, “JFF has been the search engine for the research and advocacy circles; connecting the dots so viable research has a practical outcome…The foundation has been the catalyst that has brought the research community and families together moving toward a cure.”
You all have been “our foundation”. However, there is work that still needs to be done and we are asking that you partner with us financially as we move into this critical stage of several research initiatives. Here is one of our important initiatives, Click here to view.
Our commitment to cover all the initiatives is set at 1 million dollars. This investment will be used to support our foundations programs of patient advocacy, clinical trials for a small drug molecule, and another very promising therapy is on the horizon. Additionally, the Joshua Frase Foundation has partnered with the National Christian Foundation (NCF) to allow proceeds to be given through asset-based giving. This can allow you to give through a tax bill rather than cut into long-term savings.
If you are interested in giving through wealth instead of income while reducing capital gains in tax, please contact Michael Crane at Michael@mwmlpl.com or by phone at 904.345.2047. On behalf of our foundation, our families, our researchers, and all the Joshua’s of the world, we thank you for your faith in our cause.
Wishing you and your families a blessed holiday season
~ Paul, Alison, Joshua, and Isabella Frase