The Pen is Mightier than the Denial

penSoooo…… I haven’t reached the “Let it Be” part of my last post, just yet. I tried, I took lots and lots of deep breaths, before my neuromuscular disease kicked in and I needed to stop that.

Now, I’m doing what a writer does….write.

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To the FDA (CDER Div)

Dear Sirs and Madams of the FDA,
I have a child with Rett syndrome. I am well aware that Breakthrough Therapy Designation was denied to Neuren for Trofinetide.

While, I acknowledge that I do not have an ounce of the education and responsibility that your agency has, I want to put on a personal level for you, what your denial has done.

It has crushed the souls of people waiting while their children are dying and suffering. Whatever measurements you felt were not met, let me assure you that to us, ANY improvement is significant.

Because I am my daughter’s voice (for now) I am writing for her. She participated in this trial and I do not know if she received the placebo or the actual medication, and I will not go into what I saw and heard, however, I have kept her appraised of the steps along the way, and when she heard that Breakthrough Designation was denied, she cried and said, “mad, mad, mad”.

To me, there is no more telling sign than that. My virtually non-verbal child understands and is hoping for this medication.

While, I am not naïve enough to give a thought that you may reconsider, and while I also understand the extreme responsibility your agency has to insure the safety of all, I think it’s also fair to say, my daughter was counting on you and that is a measurement that is not open to interpretation.

Respectfully,
Melinda Lancaster
and
Katelin Lancaster (Age 21)

and because I live in America, and we are blessed to be able to write to our President…..

Dear Mr. President,
I am writing to you as a parent of a child with Rett syndrome. Rett syndrome is a rare neurological disorder with a known genetic marker.  Characterized by normal development and then by a period of regression, these once normal children lose their ability to speak, use their hands, develop seizures, and a myriad of other health issues. Recently, Neuren Pharmaceuticals, a company that has developed NNZ-2566 (now known as Trofinetide)with the support of the US Army, through Intrepid 2566, applied for Breakthrough Therapy Designation from the FDA and was denied.
My daughter, Katelin, participated in the trial in Houston, TX; the results of which were used in Neuren’s application. And while I do not know if she was on the medication or placebo, I can tell you that for the first time in her life she spoke a full sentence, three in fact.
I would like you and Mrs. Obama to be aware of this drug because its possible life saving and MONETARY saving properties are tremendous.
1:10,000 girls are born with this disorder. Everyone of them will require a lifetime supply of symptom treating medications-for seizures, GI issues, breathing abnormalities, surgeries to repair scoliosis, dystonia, multiple, long-term hospitalizations, in-home nursing care. In addition, the long-term loss of income to the parents who care for them results in less of a tax base, less money going into local economies, not to mention the incredible expense to special education departments that struggle to meet the needs of students of all disabilities in this budget conscious time… well you get where I’m going with this.
I cannot say with all certainty that this medication will cure all the girls, but I feel it is possible that it will improve their quality of life, reduce the need for many of the medications, almost certainly give them a voice and reduce the quantity of care needed on a large scale, saving the Federal Government, schools, and strapped Medicaid programs millions of dollars a year.
But, in the end, it will give our children the FIRST AND ONLY hope of effective treatment. Mr. President, my daughter SPOKE to me as clear as anything. I know I can’t be sure it was the medication, but your children speak to you everyday; imagine if you had to wait 19 years for it? or NEVER? Would you try everything in your power to give them that chance to be heard? I know I would, and that is why I am writing to you today.
Sincerely and with the utmost respect,
Melinda Lancaster
USN Veteran

 

For anyone wishing to add their letters, as some other parents have already done…

Here is the link to the FDA (CDER Div) http://www.accessdata.fda.gov/scripts/email/cder/comment.cfm

To email the White House: http://www.whitehouse.gov/contact/submit-questions-and-comments

To write to the White House :

Write a letter to the President

Here are a few simple things you can do to make sure your message gets to the White House as quickly as possible.

1. If possible, email us! This is the fastest way to get your message to President Obama.

2. If you write a letter, please consider typing it on an 8 1/2 by 11 inch sheet of paper. If you hand-write your letter, please consider using pen and writing as neatly as possible.

3. Please include your return address on your letter as well as your envelope. If you have an email address, please consider including that as well.

4. And finally, be sure to include the full address of the White House to make sure your message gets to us as quickly and directly as possible:

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Washington, DC 20500

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This entry was posted in Neuren, Neuren Pharmaceuticals, nnz 2566, Rett Syndrome, Trail to a Texas Trial, Trofinetide. Bookmark the permalink.

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