Recruiting for the Myotonic Dystrophy Drug study is beginning. You must contact the individual centers to insure that you may have a chance to be included in the study. The study has only a very limited number of individuals that can be included. If you have joined one of the registries for myotonic dystrophy….this is not sufficient or enough. Call or email the centers as soon as you can and ask to be included in the drug trial if you meet the criteria.
With the correct diagnosis myotonic dystrophy can be prevented for the next generation. By using pre-implant diagnosis the next generation can be assured not to have myotonic dystrophy. This is an expensive and not practical approach in all countries though. This blog contains case reports from Pakistan of two case studies.
A recent article explored Chronic Pain and myotonic dystrophy. (click on link for full study). this is something that many of our patients experience. Heat was being used by 26% of patients. The only treatment that was relatively highly effective and was still being used by a substantial number of patients (26%) was heat. Perhaps this is because heat is an extremely accessible treatment (most people own a hot water bottle or heating pad) that has few, if any, negative side effects.
Isis Pharmaceuticals has announced a strategic partnership with Biogen to begin testing a Drug DMPKRx in Humans this fall. On a webcast that occurred on Sept 9, 2013 Isis CEO announced a strategic partnership in which Isis is receiving upfront payments of $12 Million when the start of toxicology tests begin.
The progress of Isis has been rapid for this drug. Within one year Isis identified the drug this is extremely fast. Toxicology testing program will begin this year 2013. Most likely this will be a pre-clinical toxicology tests to insure that the drug is safe in animal models. Biogen and Isis are working closely to identify bio markers and clinical testing which will begin in 2014. Bio-markers are evidence that the drug actual works to the FDA.
This fast progress is similar to what other companies are finding. As the senior researchers have said that the toxicity of the mutant myotonic dystrophy mRNA is very sensitive to these antisense drugs and other approaches.
This key announcement shows that Isis is making vast strides in this area and that human trials are set for 2014 to begin. To make sure that you are considered for the Trials:
1. Make sure that you are on the Myotonic Dystrophy Foundation Registry
2. make sure that you are on the Rochester Registry
3. make sure that you are registered with MDA in USA.
We are making amazing progress! Hope is here!
Richard
What is the cost for confirming the diagnosis for Myotonic Dystrophy. Do patents affect this cost? This Data is current as of May 2013
The costs for patents and access to testing is a major cost in the Myotonic Dystrophy Field. Here is a brief review of the costs.
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