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Wednesday, February 8, 2012

Hi Friends...

Today Congressman Ed Towns went before the House and spoke on behalf of our family and all the other families afflicted by ultra-rare diseases. Here is the clip to the 5 min speech he gave in support of the ULTRA-ACT.
http://www.c-spanarchives.org/appearance/601249385

I can't thank Congressmen Ed Towns and Cliff Stearns enough for shedding some light on our situation! Today I'm re-posting the blog about the importance of the ULTRA-act. Please read and show your support by letting your Congressman know how important this legislation is to our Nation.

Please support the ULTRA act!



As I see it there are three major obstacles that we have to overcome to save our children with ULTRA- rare fatal diseases. 1. Find the scientists 2. Fund the science-treatment 3.Get FDA approval of the treatment.



JJB has found an international team of experts working on a handful of different treatment options right now. We're funding the science and gene therapy is on its way for us. As long as we can continue to fund our research we will have a treatment in Jonah’s lifetime. The science is the easy part. 

Continued funding is an issue, but it doesn’t compare to the obstacles we will have to face once we're ready to take one of our treatments to clinical trial.

Because of the complexities of the FDA’s regulatory process, we have a very small chance of getting a drug approved in Jonah’s life time. The thought of reaching our goal of finding a drug for our children, only to be told we can’t give it to them...... Is almost as heartbreaking as the fatal diagnosis itself. 



But there is hope. In the 1990’s Congress created the Fast Track Law, to accelerate the approval process for serious and life threatening diseases during the time of the HIV/AIDS epidemic. The act did indeed accelerate drug development for AIDS and has changed its diagnosis from a death sentence to a manageable chronic disease. ULTRA-Rare disease should have been able to take advantage of Fast Track’s Accelerated Approval process, but can’t because they don’t have the prior clinical data that is needed to access Accelerated Approval. We don’t have the data, because nobody has made the effort to treat our disease, and nobody will make an effort because we can’t access Accelerated Approval. We are stuck in a catch-22 and have been deemed a lost cause.

We only know of 18 type C kids in the US and we’re not alone. There are 6,000 ULTRA rare diseases, many of which have patient populations in the hundreds, and most are fatal pediatric diseases. 

The EveryLife Foundation for Rare Diseases, along with more than 100 other patient organizations, and thousands of grassroots parent activists are the driving force behind the legislation HR3737 The ULTRA-act, which is a bill that: “Supports Accelerated Approval of Drugs for Ultra-Rare Disease to Benefit Vulnerable patients, Promote Biotechnology Innovation and Stimulate Job Growth in the United States.” The legislation details a plan on how we can drive faster cures for our ULTRA-Rare pediatric diseases. It comes down to reworking the "Accelerated Approval" process to take into consideration that our disease's come from a single gene defect that causes an error in the biochemical makeup of our children. We have good science on the disease and that should allow us to move forward with a clinical trial without having to create all the supporting clinical data that can set us back years and millions of dollars.



I can’t explain this legislation any simpler. To me this legislation is a no-brainer, it’s safe, efficient, and will help us get treatments to our dying children faster. If this legislation doesn’t pass, we must accept the fact that we may watch our kids die a slow painful death from a treatable diseases, but we lacked the political will to change status quo that prevents the transformation of science into medicine. 

So I ask you, what would you do if you were in my shoes? What if it was your kid? Would you give up and watch your child die?
I have a very small favor to ask of all of you. Follow this link http://bit.ly/supportultra and fill in the blanks, then press send. Let’s get this legislation passed now! It will cost you nothing. The choice is yours, and you can be a part of the solution.

Love & Lite
Jill ( A Phoenix Mom)