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November 13, 2013

……… i got nothin……

I’ve been home for 30 minutes, and I have cried 3 times. Nice.

Oh for my non-facebook friends…. FDA nixed the exon-skipping drug. Said it was premature and that they needed a new study.

Fuck them. They have an accelerated program for diseases JUST LIKE DUCHENNE!!!!!!!!!!!!!!!! So what the fuck is so hard that they can’t see it fits in the same category??

…………Determining whether a condition is serious is a matter of judgment, but generally is based on whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more serious one. 

Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy.
 Any drug being developed to treat or prevent a condition with no current therapy obviously is directed at an unmet need. ………………
What the fuck else do they want? My blood? My life? They can have it. Just give me a way to help my son NOW, not 5 years from now, when …I’m not going to say it. But I’m thinking it.
And now we have our 4th time….

2 Comments »

  1. Damn them.

    Susan | November 13, 2013 | 10:39 pm

  2. FUCK!

    Wendy | November 14, 2013 | 10:36 pm

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