Participant 4

Why did you enroll?  I enrolled in the study because they were looking for study participants, with very specific criteria.   I know that there aren't very many people with my type of dystrophy, in the general population, and I wanted to help future Miyoshi patents, if not myself to test out future therapies.  It's not a lot of fun, but, without patients to study, they can only model different theories without any data to find out for sure.  I hope my small part, can help, with their research for a cure. 

 

What is the purpose of the study?  The study is trying to find biomarkers to measure effectiveness for future drug or other therapy trials.

 

What did the study involve?  The study itself involved a lot of blood draws, which was no fun, but, they knew this and made every effort to make it as painless as possible.

 

The study was a week long.  They wanted me to rest in bed, for most of the time, to rest my muscles.  There were two MRI's, which were 'feet first', (to concentrate on my legs), so my head was out of the machine or just in a few inches.  The 'big event' was a muscle stress test.  They took a couple of blood draws, early in the morning, and then I was told to "run" (ok walk) as fast as I could for a few minutes.  This would tire out my leg muscles.  Then they wanted to measure my blood values for about 30 minutes. 

 

I had an IV line, so they just took my blood from it every few minutes.  From this they could 'watch' how my muscles, and how other parameters responded after stress.

 

I had a few other simple physical tests done, to measure how strong my muscles were, or how weak they had become, depending on your point of view.  It was a very thorough exam process.  Actually, much better than most of my regular Neurologist exams in the past.  I'll be going back for more testing, in a few months, so they can monitor the 'rate of decay' of my muscles and gather more data points.