Landon has a TBCD gene mutation. This is a very rare hereditary disease for which there is no viable cure.
Columbus, Ohio — As Jaren McChesney once quoted, gratitude is a miracle magnet. And Machesney and her husband, Mike, certainly now have two gratitude.
“We are overwhelmed by the support of communities throughout Ohio, across the country, and even around the world, friends, family, and complete strangers,” she said.
McChesneys has a 5 year old son Landon with a rare hereditary disease.. Mutations in the TBCD gene affect every aspect of his development.
The condition is extremely rare, and Matchesney says he only knows about 15 people in the world who have it. And that means finding a cure was a big challenge.
So they set out to find a cure for themselves and eventually Rare base, California-based public interest biotechnology company.
“Our mission is to find a way forward for you, not a dead end, for these families,” said Onno Faber, CEO of Rarebase.
To work with Rarebase McChesneys had to raise $ 50,000 by the end of 2021.. There were public petitions, fundraising activities, and even trips with a sign for College GameDay.
“It was very exciting and at the same time nerve-wracking because we were raising money, and then it became a kind of outage,” said Jaren McChesney. “And I didn’t know if it would be in time, as it would surge again.”
But with a week to spare, they did. And they couldn’t be thankful anymore.
“It’s hard to put all of our support into words, so if we’re positive, motivated, and focused, I think everything’s achievable,” said Mike Matchesney.
Achieving that funding goal meant that the next step in their journey was moving. Both Landon and his dad gave him a blood sample, which was immediately sent to California.
From there, scientists work to create neurons to test already approved drugs that they believe are effective in treating Landon.
“Without drugs related to disability, there’s not much they can do, so that’s where families and foundations are trying to intensify and sponsor research. Yes, this is what we want to help and create a platform. It’s a direct discovery platform that can benefit many people with rare illnesses, “Faber said.
This is a personal mission for the co-founder of Rarebase. He also has a rare disorder that causes tumors in his brain and spinal cord. Not receiving effective treatment for his condition, he began his path to helping himself and others.
“I have great hopes for the future. Thanks to the family working with our partners, yes, everything technically possible to bring the latest and greatest direct discovery technology. All you have to do is to deal with these types of problems, “Farber said.
Developing neurons to test takes time. You can also test whether the drug “hits” with these samples, but McChesneys already knows the next mission. You will need to raise an additional $ 75,000 to proceed with this process.
“It’s like a lunar landing to us, yes, it’s a big deal,” said Mike Matchesney. “I hope we have to find another $ 75,000. This means that Landon and other kids have viable options to deal with the TBCD mutation again, so these hits happen. It means to do. If you find those hits, it’s more than exciting. That’s another world for me. It’s very difficult to describe the possibilities in words. “
Meanwhile, McChesneys reports that Landon has a great, loving school and is advancing treatments. And the attention surrounding Landon’s journey and family fundraising allows Matchesney to connect with other families with children in that condition, both doctors and professionals who are willing to share their views. Did.
“This is what I’ve been trying to achieve over the last four years, it’s actually happening, and I’m just starting a conversation,” said Jaren McChesney. “We feel like we’re on the verge of something really huge, so we need to stay focused and keep moving forward.”
Upper Arlington family achieves fundraising goal for son with rare disease
Source link Upper Arlington family achieves fundraising goal for son with rare disease