Two boys from Bulgaria with DMD were recruited into a gene therapy (exon skipping) trial in Belgium using the TREAT-NMD Global DMD Registry. Testimonials about the process from the parents of the two boys are below:
"When our son was diagnosed with DMD, our life changed totally, we lost faith, asking ourselves why this is happening to our boy. We tried to make him feel like the others. In my opinion the registry is very useful, providing better communication with the medical specialists and providing care for our children. It gives the patients the chance for further treatment.
The trial, we are participating in, makes us very happy and gives us faith, even if the trips to Belgium are long and tiring. Everything is very well organized and my son feels very secure in UZ Ghent. I hope that soon there will be a cure for all the boys with DMD."
"We appreciate the TREAT-NMD registry very much, as it provides information to the specialists on the number of the affected boys and their precise gene mutations. This registry helped our boy to be included in a clinical trial in UZ Ghent and we really hope that this treatment will have a positive effect on him. We want to thank the doctors in UZ Ghent, because the organization of the trial is very good.
Hopefully new treatments will be available to all the affected patients. We are very pleased to our doctors, for their care. They inform us for everything new, concerning the disease of our boy. I want to thank to the doctors, patients' organizations, scientists and pharmaceutical companies that are making efforts for finding a cure for DMD."