How Little Liam is Doing 11 Months After Receiving EDI200 in Clinical Trial
Liam is a happy little guy whose smile lights up the room – and your heart. Like most 11-month-olds, he drools constantly and often has a runny nose. Unlike most kiddos his age, that saliva and mucous is a welcome surprise for his parents.
Liam is affected by x-linked hypohidrotic ectodermal dysplasia (XLHED) which can affect, among other things, the nasal glands, causing nasal concretions, and the salivary glands, causing dry mouth and swallowing difficulty. When he was just days old, Liam participated in the Newborn XLHED Clinical Trial and received five doses of EDI200, a lab created protein that is missing in people affected by his condition.
It is too soon to know if the EDI200 did indeed have a positive impact for Liam. But his parents, Steve and Beth, are grateful that Liam’s glands seem to be working correctly. Who knew we could be so excited about snot and spit?!
The family was at St. Louis Children’s Hospital this week for Liam’s one-year checkup as a part of the Extension Study for the Clinical Trial. We, the NFED, announced earlier this month that the Clinical Trial didn’t achieve its expected outcomes, and therefore was not continuing at this time. While it is certainly disappointing, we are grateful that Edimer will collect data for 10 years on the 10 babies through this Extension Study.
Liam’s dad, Steve, shares our hope for what this data could tell us.
“It’s going to be four to five years before we will know about the number of teeth these babies will have,” Steve said. “We might yet learn that EDI200 did impact their dentition. We must stay the course. We are not giving up, for Liam’s sake and for his kids and the next generation.
“It’s what hasn’t happened in the Clinical Trial that is just important as what has. Like in the animal studies, there has not been one negative side effect for the babies who received EDI200. That’s important and advanced research,” Steve said.
Beth said, “I admit that I was disappointed when I first learned the news about the Clinical Trial. I was thinking, ‘Why are we stopping now? What does this mean for Liam and his future?’ But it doesn’t change how we will raise Liam.
“In our case, we are better off having participated since Liam does get runny noses and has saliva. His skin has been normal. He has not had any respiratory issues that are common in XLHED, and his nails are good. He’s tall. He’s underweight for his age but we know that’s typical of the condition. He has not had any teeth erupt yet but the dentist thinks he will have six baby teeth. Given what I lived through with XLHED, my greatest hope is that Liam will develop teeth. These are all things which have us hopeful for the future,” Beth said.
Both parents say their participation was always about the bigger picture: a research breakthrough for XLHED.
“We had the opportunity to help advance research in a health way. We embraced that opportunity,” Steve said.
Beth agreed, “We will continue to advocate and support efforts from families, the NFED and Edimer to keep this research at the forefront.
Steve said, “We love that Liam is a part of what ultimately will be a solution. It’s pretty cool that one day, he could say to his kids, ‘I did that for you.’”
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