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.

This is a phase 1 study checking for the safety of the drug. Is the drug safe in humans and what levels of the drug will be present in the blood after a period of time?? These are the questions that this phase of the study will look at.

Its important that drugs be safe and that they work. It would be against ethical principles to do a large scale study of the drug until the safety aspect is known. Most drugs have some side effects and these need to be known before it can be presented to a larger population or larger number of people to test the drug.

It is also important for researchers to know how much of the drug is in the body and how long it stays there. Your body is generally pretty good at getting rid of foreign bodies including drugs. So how long does it take the kidneys, liver or other organs to excrete the drug? This allows the researchers at ISIS to know how much to inject and when to inject the drugs.

There are well over 30,000 people with DM1 in the USA. Only 18 will get the Drug. 18 others will get sterile water injections. These golden 18 will be the ones that decide if the drug moves forward to phase 2. The drug companies and researchers will carefully screen all patients to meet the criteria. It is critical to make sure that you meet all the criteria so that the drug study can go on to Phase 2

The Golden 18 will be the brave and lucky to try the new drug. Lucky as many will want the treatments. Brave as it is a new drug and could have side effects.

Into the future we go, braving a new path forward, The  “golden” 18 light the path, carry the torch for hopefully all to follow.