This is my first diary, and I hope that my second diary will be to thank the community for saving lives. Although I don't have time for politics, liberal or conservative, I am told that there are thousands of people of good will on this site, and perhaps even some veterans of the campaigns that once forced the FDA to expedite the approval of AIDS drugs rather than letting the disease continue to claim lives. I am asking for your a few minutes of your time, lobbying the FDA to reverse a cruel, bureaucratic decision, one undoubtedly influenced by large pharma, which has its own agenda.
Duchenne Muscular Dystrophy is a horrific disease that gradually makes the muscles worthless, leaving afflicted boys (and very few girls) wheelchair bound until eventually they are incapable of even coughing and finally can't breathe. Until now there has never been any hope to offer the DMD boys (you might know them as "Jerry's Kids"). However, recently, a new drug called eteplirsen has proven miraculously effective in small trials. The mothers of the children in the trial have advocated tirelessly for early approval of the drug. They know it works--they see this every day--and see no point in delaying access to other children while the FED puts up endless bureaucratic roadblocks to final approval. They have enlisted the aid of dozens of representatives and senators, such as Elizabeth Warren.
Despite incontrovertible scientific evidence that the drug will provide hope to DMD boys for the first time, the FDA recently announced that it wants to spend another 2-3 years collecting additional data before making a final decision. Understand: If this wrongheaded bureaucratic process (the same one that was overthrown by tireless AIDS activists many years ago) is followed, several hundred boys will lose the ability to walk every year, and several hundred young men will pass away, unable to breathe, while their mothers hover over their bed, unable to help. I am not overdramatizing the situation. It is very real. DMD might not be as common as AIDs, but those boys deserve the same support nonetheless.
The DMD parents are organizing letter writing campaigns and discussing camping out on the FDA lawn in protest. Please support them. I am providing a link that includes FDA e-mail addresses and a template of a letter you can write (or use your own words, if you feel that is more effective). The link also includes a fuller explanation of the situation than I have written here.
charley's fund letter writing campaign
Thank you so much!