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

Review information for Patients With Myotonic Dystrophy

So you’ve been touched by Myotonic Dystrophy. You need a lot of information and Help. The Myotonic Dystrophy Foundation is the premier foundation working to advance the cause and help define treatments. They have a great toolkit to help with a broad understanding of the disease.This toolkit is 124 pages long. Its great!

The Muscular Dystrophy Association of the USA is also working hard to cure the disease. Most Myotonic Dystrophy Patients visit the MDA clinics from Time to time. MDA USA funds a lot of research and they have a new in focus information on Myotonic Dystrophy. Focus on Myotonic Dystrophy MDA USA. There are about 30 pages in this compact review of myotonic Dystrophy. A nice review and graphics of the cause and treatments of DM1.

MDA Issues Winter Research Grants

The Muscular Dystrophy Association of the USA (MDA) announced its winter research grants. 13.6 million was awarded including two grants for Myotonic Dystrophy research. One grant to doctor Disney will explore the possible therapeutic effects of small molecule approach for DM2. This would be a strong complement to the DM1 research that is ongoing in his lab.

The other grant to Dr. Mockton is to research the effect of Triplet expansion and stability on the likely course of the Myotonic Dystrophy Disease.

MDA is spending 4.6 of its research grant budget this winter on Myotonic Dystrophy Research.

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Help From MDA Clinics

Many people affected with Myotonic Dystrophy get help from the MDA the Muscular Dystrophy Association. They have clinics all around the United States which are accessible to all even those without funds. They can also help with wheelchairs and other resources. They run summer camps for kids which is awesome as well. Here is a video that  a couple that is affected with myotonic dystrophy has made about their experience with MDA

 

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Trip to the MDA Clinic

Each kid in the USA gets to go to the MDA clinic a couple times per year. These clinics are great and allow you to meet with Dr’s and Nurses. The MDA pays for the visit if you do not have insurance. They also help with payment for stroller, wheelchair, etc. if you do not have the money to pay for this.

MDA Clinic at California Pacific Medical Center in San Francisco, CA USA (415)921-2101

At the Clinic you will meet with:

  • Nurse who takes Blood Pressure weight etc.
  • Doctor who checks on physical well being
  • Social Worker
  • Physical Therapist
  • Occupational Therapist
  • Speech Therapist
  • MDA Representative
We usually go to clinic once or twice per year. It helps to give some perspective. and to see what is going on with the MDA and get the knowledge of these workers.

We usually go to clinic once or twice per year. It helps to give some perspective. and to see what is going on with the MDA and get the knowledge of these workers.

Chris does not like to go to Doctors and Hospitals etc. So he usually is a little upset at first. But then he calms down. We usually bring his radio or tape recorder and this calms him down a bit.

Here is Chris and Dad at the Clinic. Chris really likes his radio and we have tape and book sets that he really likes.

Chris can entertain himself with the his radio and books quite well.

Here is Chris with Aunt Lori in the waiting Room. My sister helps me a lot with Chris. Thanks Lori!

Here is Dr. Mednick at the Clinic. He is great and spent a lot of time with us. He has been with MDA for a very long time and knows his stuff. Very good with patients. He’s only there two days a month so make sure you go when he is available. Dr. Mednick phone is (415) 921-2101 FAX (415) 457-0602

Here is one of the great therapists. She was very helpful. See the pink tube. This is a nice toy that Chris really liked. It helped focus his attention

So that’s the good. What you should not expect is that the Clinic will give you a lot of new information. If you’ve read this page you probably know more than the staff about this condition. Be proactive. We got a 12 lead ECK done with Chris when we were there last. It helps to have all the tests as a base line so the Doc’s can know what to expect. Some people have reported not good experiences at their clinics. We have been to three different clinics. One in Rockford, IL USA, One in Chicago, IL USA and this clinic in California. Each one does things a little different. The California clinic was different in that that had a knowledge doctor who spends time with you. The head of the Clinic Dr. Miller is great but really busy. He spent about 3 minutes with us versus the 50 minutes with Dr. M. So you can tell this clinic is sharp as they take care of patient needs in a very effective manner. You’ll find each clinic a little different… We’ve found that clinics in rural area’s are not as knowledgeable. They probably see less patients and therefore do not know as much. Help to educate them and give them this web site. Don’t expect them to read it though. Dr’s are very busy and have little time for education when real patients call. If you find one with knowledge make sure to stick with them and let me know. I will post their name on the site.