Author Archives: Richard

About Richard

Inventor, entrepreneur, father, medical device manager.

Drug Testing for Myotonic Dystrophy Drug Starts this Fall

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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

Myotonic Dystrophy Patients Show Vitamin D Deficiencies

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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.

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Isis Pharmaceuticals Myotonic Drug

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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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Myotonic Fundraiser Tweet #raceforall

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A retired surgeon from Bosham wants to raise as much money as possible for the Myotonic Dystrophy Support Group in England in support of three family members who suffer from the condition when he once again takes part in the J.P. Morgan Asset Management Round the Island Race. Tweet #raceforall to raise 10p ( about 16 USA cents)