Clinical Trials and Duchenne
While there’s still no cure for Duchenne Muscular Dystrophy, we certainly understand far more than we did 20 years ago. We also have more treatment options now.
All that was accomplished with the information we gathered from clinical trials.
Unfortunately, actually participating in clinical trials is definitely a mixed bag. It can be quite challenging for a family to decide if the rewards outweigh the risks.
On one hand, there’s that carrot dangling in front of you… that hope that your loved one with Duchenne gets the actual drug in the trial instead of the placebo, and that it maintains or reverses your loved one’s current condition. That hope that your loved one accesses it years before it’s available to the masses. That hope that your loved one might live longer or have a better quality of life because of This. Specific. Trial.
On the other hand, the burden on the family can be huge. Between frequent travel, the time commitment, medical procedures, separating the family, (and so, so much more), every single person in the family pays for the opportunity for the individual with Duchenne to participate.
Enormous responsibilities and decisions accompany life with Duchenne, and this is just one of the many: our entire Duchenne community – spanning across countries and time – will benefit from the clinical trials, but how much are the “lucky families” that are accepted into trials willing to sacrifice in order to participate?
It’s quite a heavy decision that many families must make – extreme personal sacrifice in exchange for potential personal gain and potential community gain. The hard truth is that if no single family makes that personal sacrifice, then our entire community will never have the chance at accessing better treatments.
ClinicalTrials.gov is where nearly all clinical trials in the United States are listed. Please take some time to browse through the website to see how many clinical trials are available for DMD, to learn a little bit about eligibility/screening and also what happens once a child is accepted into the study.
Thank you to all our families who have participated in clinical trials...
