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My son’s time is running out due to
a rare disease. The FDA needs to add
more clinical trial flexibility

Original Article

Posted By: Daria, 2/28/2023 3:01:21 PM

My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the type known as CLN3 — is stealing his childhood. And then this rare disease will steal my child. Wheeler is missing the DNA needed to recycle a waste product called lipofuscin that his cells naturally produce. As lipofuscin builds up, his condition will get worse, robbing him of his eyesight, his speech, his mobility, and ultimately his life. Our only hope is finding a treatment to cure, or at least a way to slow down this unrelenting disease.

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Reply 1 - Posted by: Island Life 2/28/2023 3:22:11 PM (No. 1413983)
I'm sure Ms. Stecker is aware that when Donald Trump was President, he worked on making experimental drugs available to people who had nowhere else to turn, they had exhausted all options. It looks as though she has pretty much gone that far. This is not the time to be sitting around discussing the difficult situation. I can only suggest she somehow gets in touch with President Trump and get his contacts and thoughts on the issue. I wish you the very best Ms. Stecker that your little boy gets the help he obviously needs. And as an aside to you, I smiled reading the end of your article. My little girl was born with a severe congenital heart defect in 1975, her birthday being February 27th, yesterday. It's good to know that this most important day to me now has a good anniversary to match.
18 people like this.

Reply 2 - Posted by: Jean123 2/28/2023 3:42:17 PM (No. 1414002)
If insurance won't cover the cost of the medication, can't she start a gofundme or givesendgo to cover the cost of the drug?
5 people like this.

Reply 3 - Posted by: seamusm 2/28/2023 3:53:11 PM (No. 1414009)
Public calls for cost-sharing might work. I worked for 30+ years doing university-sponsored drug trials for largely one particular rare disease and fully understand this mother's frustration. But over that same time, I have been repeatedly frustrated by the ever-increasing bureaucratic impediments that seem to be just ways to demand hiring more federal employees and guarantee deep-state jobs. Caring is just lip service and money is the ultimate power.
6 people like this.

Reply 4 - Posted by: DVC 2/28/2023 4:56:29 PM (No. 1414053)
I thought that there was a "right to try law" which Trump had passed which would let people try any drug that they want if they are in an incurable situation. That there are people who would prevent an attempt seems to be just cruel.
14 people like this.

Reply 5 - Posted by: MickTurn 3/1/2023 10:22:25 AM (No. 1414649)
The FDA is all about the revolving $$$$ door with Big Pharma, don't think for 1 second they are there to help anyone.
1 person likes this.

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My son’s time is running out due to
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more clinical trial flexibility
5 replies
Posted by Daria 2/28/2023 3:01:21 PM Post Reply
My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the type known as CLN3 — is stealing his childhood. And then this rare disease will steal my child. Wheeler is missing the DNA needed to recycle a waste product called lipofuscin that his cells naturally produce. As lipofuscin builds up, his condition will get worse, robbing him of his eyesight, his speech, his mobility, and ultimately his life. Our only hope is finding a treatment to cure, or at least a way to slow down this unrelenting disease.
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