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My name is Julie Ogden and I have a rare form of muscular dystrophy (Hereditary Inclusion Body Myopathy [HIBM]. Recently, the gene that is defective in my muscles was isolated. It is a single gene and it's pretty small. About three years ago, An Oncologist, who established the Mary Crowley Medical research Center (MCMRC)in Dallas heard of my case and believed he had the technology to insert a healthy gene into my muscle cells to replace the damaged gene. Since that time, through various friends and family, we were able to raise the money to start working on creating a mechanism of transport to insert the healthy gene into my muscle cells. August 19, 2008 was the day I received the first experimental injections of healthy gene into the muscle cells of my left bicep and wrist muscle. Subsequently, I have received three further experimental injections in my right wrist muscle.
So far, I have experienced no side effects from the experimental injections besides a low grade fever and some facial flushing which only lasted approximately 60 minutes. The strength in both my left and right wrists has improved even though the dose of gene therapy I received was only 1/100th of the actual therapeutic dose needed to increase muscle function.
Our future plans include administering the experimental gene via IV infusion. We are waiting for funding for FDA testing required to begin this investigational treatment. Once we find the proper dose of investigational agent that can be given with the fewest side effects, we plan to begin a nine patient phase 1 trial.
Our biggest delay right now is finding the money to go forward with the research. The Neuromuscular Disease Society (NDS) along with my family and friends have been our biggest contributors so far. However, we are still lacking funding. A copy of the budget of money we require is displayed on the ‘Donate’ page of this website.
It is amazing to me how much has been accomplished in this short period of time. God has definitely put the right people, in the right place, at the right time to make a cure possible for everyone who suffers from HIBM and muscular dystrophies.
ATTENTION!!!!!
August 14 2009
Dear everyone,
I went to Dallas yesterday for the day 30 muscle measurement. I have not yet received the statistical results from the dynamometer. However, I can write that I have improved in some muscles while others have remained stable. The muscles that have improved are my left wrist, left and right quadriceps, and toes. Other muscles such as my fingers, and grip have improved even though they are still not measurable on the machine. I'm very pleased with these results as this dose of gene therapy was really too small to show any efficacy. I leave in the first couple of weeks in September for the second dose which will be increased. I will definitely let you know how it goes. Thank you for your prayers!
Love,
Julie
"The Lord has done great things for us, and we are filled with joy"
Psalm 126:3
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