Where’s the Beef? Good News Bomb coming?

Okay lots of hype on the Isis Drug that was supposed to be in early stage trials this year in actual human beings. To us waiting January 1st is a good day to start. Now we are in JUNE! Yo Isis! We are waiting patiently.

I would imagine (hopefully) the results of the lab test have been so incredibly positive that they had to delay a few months to contain their joy at what a great drug they developed………. Hopefully not the converse.

We are waiting Isis for the good news. Drop us a good news bomb!

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Chronic Pain and Myotonic Dystrophy

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.

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Drug Testing for Myotonic Dystrophy Drug Starts this Fall

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

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Isis Pharmaceuticals Myotonic Drug

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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Isis Myotonic Drug Considered for Autism Accelerated Trials

The fast fail initiative is considering many potential autism drugs including Isis Pharmaceutical’s pending drug to treat myotonic dystrophy. This  “FAST FAIL” initiative funded by NIH looks at promising drugs to treat Autism and several other conditions. It enables the drug to go to human trials earlier and see if the drug will pan out. The Isis drug by far is the most probable drug to treat and reverse the course of the Autism disease.

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