Our Mission
Our goal is to speed up the therapies that keep DMD boys as healthy as possible until a cure for Duchenne Muscular Dystrophy is found. These disease-modifying therapies (DMTs) can aid in the preservation of the muscle cells of DMD boys. Because injury is ongoing in the muscle cells of all DMD boys, our mission is a race.
DMD Therapies
The lack of a functioning dystrophin gene prevents production of the dystrophin protein in the cells of a DMD boy. Dystrophin is necessary to many fundamental life support functions in a muscle cell. A systemic cure for DMD requires restoration of the dystrophin gene to all muscle cells in the body. This gene therapy, while theoretically identified, is many years away. But therapies that can slow the progression of this disease are attainable now.
Read more...Compounds of Interest
1. L-arginine and Deflazacort
2. Disodium cromoglycate
3. Magnesium (a calcium ion antagonist)
4. S107 (affects ryanodine receptors)
5. Debio-025
Why Now?
If you are the parent of a DMD boy who is experiencing irreversible muscle loss daily, the scientific debate highlights an unacceptable proposition: maybe we will get it right. If more is possible, it should be done. Impediments must be removed. But how? What agency can guarantee that the right therapy will be developed in the fastest possible time to reach and help the most victims? Who conducts that research? There is no single authority that can answer that question. We do know that new research methods can dramatically help assess competing therapies. The proof is in the pudding, so to speak. And (to extend the metaphor) the pudding can now be inspected more effectively and in greatly shortened time frames.
Read more...Whatever it Takes
When possibilities become probabilities and new research methods provide faster pathways to medicine, it is time to act. That action has to be fail-safe. Any possible interruption of the pursuit of compounds to maintain DMD muscle cell must be eliminated.
Read more...Bench to Patient
The journey from research to medicine is highly vulnerable to funding and manpower interruption. Only large pharmaceutical companies can vertically integrate and go bench-to-patient, but the DMD patient population is too small for commercial medicine development. That means research that demonstrably reduces DMD muscle cell damage simply stops without a "sponsor" who will take it to trials and then medicine.
The University of Minnesota medical school has been able to put utrophin, a close relative of dystrophin, into cells using a cell-penetrating tag called TAT. The utrophin protein with TAT avoids immunity causes and can reach every cell in the body, and the researchers are "hopeful the therapy can move into human clinical trials within three years."* Can three years be shortened? Can the conditional word "hopeful" be changed to "proceed now"? The RaceMD mission is to compress time and eliminate barriers, when that is humanly possible.
Read more...New Research Methods
A breakthrough in testing methods allows human DMD muscle cells to be grown outside of the human body. This allows direct testing on human muscle cells lacking dystrophin. There are other breakthroughs.
Compounds that can work have been documented and deserve immediate testing. FDA approved compounds have shown beneficial results and should be tested and trialled.
Read more...Boys and Parents; Their Stake
Boys and their parents are entitled to know what is in testing, by whom, and for what objective. For test processes identified here, parents are entitled to vote on their preference for further testing, compounds and trials.
Read more...Whatever it takes
