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We'd never heard of Pompe disease. Aileen and I are silent carriers … but it doesn't manifest until you have a baby with somebody else, and even then, there's only a one in four chance, so for us, the odds were very low.
Found on FOX News 8 years ago
Those were six weeks in the ICU that we'll never forget, but she fought through it, and I realized that that fight in her eyes -- that she didn't want to give up -- and we knew then, that if she wouldn't give up, then we wouldn't either.
Found on FOX News 8 years ago
We didn't know that we could change the course of their disease, but we wanted to never have any regrets that we didn't try to do everything we could, ultimately, that's what we did, was to help move science forward and bring attention and resources to the disease.
Found on FOX News 8 years ago
What the enzyme is designed to do is to replace the enzyme that's missing or defective in people with Pompe, it's designed to get to muscle cells, including the heart, which is a cardiac muscle, to get to the diaphragm, which is involved of course in breathing, and restore muscle function as best as it can.
Found on FOX News 8 years ago
I've met many moms and dads who continue to be not just great champions and advocates for their children, but tremendous entrepreneurs and having that patient perspective is so vital, i think having that patient perspective is probably what sets Amicus apart from our peers. Many great biotch companies are patient-focused, we try to do it to an extraordinary level here.
Found on FOX News 8 years ago
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