Another mom from etep trial tells her story…
Posted by: Christine McSherry on December 26, 2013
From Mom – Kimberly Procko, her son; Evan is in the Sarepta trial, more evidence that the drug IS working. There is no need to argue over endpoints, or dystrophin – for the first time ever, there is a drug that is slowing the progression of Duchenne. There should be nothing slowing this community down to get access for those who need it, nothing. The ENTIRE community needs to stand behind Duchenne – and make the FDA realize that Duchenne is a priority, it is acute and kills. Duchenne is not a “chronic disease” that has variable rates of decline. When those affected begin to decline – they don’t stabilize. The only disease community that has ever changed the way FDA does business is AIDS. Dr. Woodcock said it herself during the opening for the Policy Forum, the AIDS community created change…
I suggest we create change as well – if we don’t, we stand the chance of standing in the way of a treatment getting to our kids.
“Evan cried when he looked at the surgery from his third biopsy. He’s wants to pretend he’s normal and didn’t want a third scar to hide from his friends. But he did the third biopsy because he trusted us. It would be the one to prove the dystrophin was present. He has and continues to have night mares of hospitals and painful needles. This trial was a chance for extended life for all at the expense of quality of life for over two years now. Things have been better since the port but still a couple gut wrenching painful mishaps with missing the port have occurred recently. But I know it’s working. He hasn’t had to wear night splints for over a year. He has no contractures at night like before and has lost no flexibility. His calves are always soft and his fists are no longer clenched at night. He no longer needs tons of prunes, oatmeal, and Miralax to go potty. He goes everyday on his own with not much attention. He is not cured but I believe the progression has slowed and in some ways improved. I believe he can play the drums and dance because of Eteplirsen. I would be devastated if these small but significant things were taken from him. That is what tortures me. I am not mean spirited. I don’t like this. I can forgive and even admit mistake. I am hoping that I can prevent the worst thing from happening by bringing these issues to light. I can’t forgive my self for silence. Can any of you? I don’t want 20 /20 vision looking back with regret.
12 hours ago via mobile · Like · 5
Kimberly Procko, I agree and have been saying it all along, with Jenn Mcnary, Mindy Schneider Leffler and Tracy Kramer Seckler - These moms – will fight harder in 2014. (and holidays just get us more fired up)
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