Author Archives: Richard

About Richard

Inventor, entrepreneur, father, medical device manager.

ISIS Issues Press Release on New Myotonic Dystrophy Drug

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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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The “Golden”18

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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.

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

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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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Extraordinary Gift will advance DM2 Research…. WOW!

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Most of the emphasis on myotonic dystrophy has been on DM1 or Myotonic Dystrophy Type 1 Now a new generous gift will push the reseach front on DM2! A great day!

 

Gift Will Advance Research on Myotonic Dystrophy Type 2

September 09, 2014

 

 

Generous Donors!

Albert (Alfy) and Lilyan (Lil) Nathan

A $1.25 million gift from Lilyan (Lil) and Albert (Alfy) Nathan of Florida and Michael and Sherry Goldberg of Chicago will create a new center dedicated to research on myotonic dystrophy type 2 (DM2) at the University of Rochester School of Medicine and Dentistry.  The gift will be used to support a new research program that will be led by UR Medicine neurologist Chad Heatwole, M.D.

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ISIS Announces Phase I trials For Myotonic Dystrophy Type 1 Drug

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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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