ISIS Issues Press Release on New Myotonic Dystrophy Drug

ISIS Pharmecuticals announces testing of new Drug to Treat Myotonic Dystrophy.

If you have this disease call you local research center and ask to be added to list of potential patients if you want to participate.

Isis Pharmaceuticals Initiates Phase 1/2 Study of ISIS-DMPK Rx in Patients With Myotonic Dystrophy Type 1

ISIS-DMPK Rx Extends the Targeting of Antisense Drugs to Include Muscle Tissue

CARLSBAD, Calif., Dec. 16, 2014 /PRNewswire/ – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a study for ISIS-DMPKRx in patients with Myotonic Dystrophy Type 1 (DM1).  DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells.  ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA.

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Full Phase I Drug Trial Recruiting for Myotonic Dystrophy (DM1)

This week ISIS pharmaceuticals announced the commencement of recruiting for Phase I of the Myotonic Dystrophy Drug Trial. This is tremendous news for the myotonic dystrophy community. The promise of scientific research to define the DNA site, identify the cause of the disease,  then begin to develop treatments that would reverse the problems in the cell are culminating  to the promise of hope for all that suffer from this horrible affliction.

If you want to participate in this study you must act quickly and contact the centers that are conducting the research. Find the closest location and contact the person via email or phone. You must meet all (meaning all) the qualifications for the trial. The full information is on CLINICALTRIALS

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ISIS Announces Phase I trials For Myotonic Dystrophy Type 1 Drug

Happy Days! A clinical Trial is going to start on Phase one of Myotonic Dystrophy Drug. That means that a small number of patients in the US usually under 50 will get a chance to try the drug. The purpose of the Phase I trial is to

Try out the drug
Find an optimum dose
Assess Safety of Drug

More on what a phases of clinical testing mean  (From MD Anderson site)

What are Phase 1 Clinical Trials?

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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!

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