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, 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.
On the other 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.
And the clencher? Our community will never access better treatments or a cure if families don’t participate in clinical trials.
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?
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 every single Duchenne family who has ever participated in a clinical trial!