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We Need The FDA To Lower The Age Limit For Gene Therapy Before It's Too Late!

This petition had 104,773 supporters

When I learned one of my closest family friends was going blind, I wanted to help. Gary is 16-years-old, three years older than me, and we’ve grown up together. Gary needs a treatment called gene therapy, but the FDA says he has to wait 2 years until he is 18. By then it will be too late. I started this petition because he is like family to me and little did any of us know, we’d be fighting to save Gary’s vision together.

Gary’s vision is almost entirely gone in his right eye and rapidly declining in his left eye. This happened almost overnight. Gary saw a lot of different doctors before he was finally diagnosed with Leber Hereditary Optic Neuropathy (LHON). LHON is a rare disease which causes sudden, irreversible blindness. At this time there is no cure, and he can no longer do many of the things he loved to do, such as play baseball and read.

While there is no cure, there are treatments that may be able to stop the disease from progressing and possibly even restore vision. One of the most successful treatments is gene therapy. Gene therapy takes a correct copy of a gene and adds it back into the cells that have a mutated version of that gene, which fixes it. This treatment is new, and is in the clinical trial phase. Though Gary is a perfect candidate for these trials, he is disqualified due to his age.

GenSight Biologics is currently running a gene therapy trial for Gary’s mutation in which patients must start the therapy within one year of symptoms. The FDA has not approved this trial for anyone under the age of 18-years-old. When he’s old enough to be enrolled, he will no longer be eligible for this trial and it will be too late. The FDA has approved other gene therapy studies for children; some trials include kids 10-years-old or younger, so it does not appear to be a safety problem. This just doesn’t seem fair.

I want Gary to be able to see again. He has his whole life ahead of him, which is why I am asking the FDA to reduce the age restriction to 16-years-old for GenSight Biologics Phase III Clinical Trial, GS010 in Leber’s Hereditary Optic Neuropathy (LHON). This is Gary’s only hope right now. Please join me and sign and share this petition.

Thank you.

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