RESOLARIS - THE CURE FOR FSHD?

As I have mentioned in my previous posts, there are no current approved medical treatments that cure any type of muscular dystrophy.  There is truly exciting news  about a clinical trial for Resolaris, a potential solution for people with facioscapulohumeral muscular dystrophy (FSHD).   FSHD typically starts in the face and upper body and progresses to the lower body.  People with FSHD typically experience debilitating fatigue and chronic pain.   FSHD can be fatal, especially with those who have an early onset.  FSHD is typically diagnosed by the pattern of muscle weakness, DNA testing and other symptoms.

Resolaris is a new drug developed by aTyr Pharama,  a San Diego based company.  Resolaris is derived from protein released into the blood by human skeletal muscle cells.  Resolaris is administered intravenously.  aTyr believes that Resolaris will provide help to people with rare myopathies with excessive immune cell involvement.

The clinical trial is the first administration of a naturally occurring protein derived from physiocrines.  Pysiocrines are a new class of physiological modulators that promote the process of restoring stressed or diseased tissue to a healthier state.  The clinical trial is scheduled for 44 FSHD patients in the European Union.  The trial is designed to evaluate the safety, tolerability, pharmacokinetics and biological activity of adult patients.

For more information about Resolaris please visit the aTyr websitehttp://www.atyrpharma.com.

PERCEPTIONS

People have perceptions that doctors know everything about muscular dystrophy.   A general practitioner will not be as knowledgeable about the disease due to the broad variety of issues that they deal with.  If you have any questions that you might possibly have a form of muscular dystrophy go directly to a neurologist who deals with muscular dystrophy and the related diseases.

Obviously, neurologists who specialty is neuromuscular diseases do know quite a lot about the various types of muscular dystrophy.  Actually, they are quite adept at diagnosing this disease.

Since there are no known cures available, I need to outline that this is your medical issue.  This is your fight, obviously you doctor can help, but you need to take the responsibility to beat this disease……no one else can do it.

This disease will beat you or you can beat this disease.   In other words…..blaze your own trail.  Do the things that are going to help you deal with this crippling rehabilitating disease.  Don’t believe that there is nothing that you can do to help you live with this!

DEPRESSION

Depression impacts nearly everyone that has muscular dystrophy (MD).  Yes, most of us have to deal with it since MD impacts our lives so significantly.  Once you understand that you have this disease it is difficult to deal with the reality.  Especially, once you understand all the ramifications and that your life will never be the same again.
   

When I was informed that I had muscular dystrophy, it was as if I had been run over by a truck.  The doctors indicated that FSH runs in families.  So, I thought that the doctors had to be wrong again (denial). I am the exception to that rule, as I did not have FSH or any other type of muscular dystrophy on either side of the family.

That is when depression set in for three days…it was like a black hole that I could not get out.  I was depressed about the difficulty of the situation.  Then, I decided that I was going to find a solution and BEAT this disease.  So, I started researching MD and any possible solution.

So, the first thing to do is to recognize it for what it is!  Here are some of the classic symptoms.

1.    Feeling of sadness

2.    Feelings of worthlessness

3.    Lack of interest

4.    Lack of focus

5.    Thoughts of suicide or death

 SUMMARY:

Depression is a normal emotional condition with any major disease.  If it becomes more pronounced you should seek competent medical care.  I chose to not discuss it with the doctor or take any type of medication.