The DMD Community Needs Hope Again

Simon was such a happy baby,he flashed his one dimple on his second day of life, while he dreamed of a life so sweet. Skinned knees, bruised shins and forehead, terrifying tumbles down the stairs, but the tears never lasted long, he was one resilient boy. At six his problems were given a name: Duchenne. It seemed like a nightmare that we could not wake up from. Slowly, agonizingly we came to the realization that there was nothing we could do to stop the train that was barreling down the track towards our beautiful son. We would help him make it the best life possible. Sure, we'd check on the latest research, cut our articles for the doctors, just in case. But with no expectations of a treatment coming in time to save our child from the severe ravages of DMD. And then one day we got a call, and we enrolled our son in a clinical trial for exon 51 skipping, but again, without any expectations, and knowing that he might get a placebo for one whole year's worth of weekly injections. Slowly, despite our reservations to allow hope to cloud our judgement, we saw stabilization, we saw little improvements. He stopped falling, he could hop a bit, he could skip for the first time in his life, he could ride his bike again, something that he had not been able to do in the 2 years before the trial.His medical team was speachless.The trial became a routine, part of our life. And then the rug was ripped out from under our feet as the Drisapersen trial was halted.

The progress we saw in our son and others, the hope it brought was gone. It has been 6 months now with no drug, 6 months in the pergatory of waiting. The train is back and the track leads to our door. Only the FDA has the power to switch the track on DMD by approving the drugs that come available, without wasting more time with placebo trials. Right now it's Eteplirsen and Drisapersen, hopefully very soon other treatments to help all with DMD. The natural history of DMD is well known, as is our tolerance for risk with such a devastating disease. Please, help us to buy time for our children until a cure can be found. The DMD community needs hope again, not bitter disappointments.

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