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
A recent Italian study of myotonic dystrophy patients concluded that many patients up to 88% may show deficiencies of Vitamin D. Over 40% of patients showed severe Vitamin D deficiencies. Patients with DM may need to consider a multivitamin of Vitamin D supplement to insure adequate nutrition. Many DM patients have less social and ambulatory abilities which may limit their outdoor activities in sunny weather. The sun which is a source of vitamin D.
Informal reports continue to give good vibes on the drug that Isis pharmaceuticals is developing for myotonic dystrophy (DM). The drug is an antisense approach one that will break up the clogs that are causing the DM disease. Very little information is being released by the company, however.
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