2-year-old, friends battling lethal form of dwarfism hope for breakthrough in clinical trial

CHARLOTTE, NC (WJZY) - Two-year old Jude Peters is one of less than 60 kids in the world fighting a lethal form of dwarfism.

"Things that most people would never even think about, Jude is dealing with them every day and he's overcoming them every day, " said Jude's parents.

He’s not alone.

Six-year-old Marley Bedford was born with rhizomelic chondrodysplasia punctate, the same disorder as Jude and all of the other kids meeting here at children's harbor in Alabama for a conference.

The thing is, Marley is doing things the others cannot

"Well, Marley is wild, she's very just tenacious and crazy and into everything and she's just full of life," said her parents. "The only difference in Marley and the other kids is her level of plasmologens."

All humans have plasmalogens, but the distinction of rhizo kids is they have very little. Marley is the exception. She has more than Jude, but less than the average human.

"A normal level of plasmalogens the number in the body would be .67 or somehwere around there. Classic kids like Jude tend to run closer to the .01. Marley is .36."

Baby Jude and other rhizo kids are scheduled to start a clinical trial this fall in hopes of raising their plasmalogen levels. Doctors aren't sure what type of results they'll get but families are hopeful.

"The hope with the treatment overall would be if we could get Jude's plasmalogens to where Marley's are at or someone like Marley that Jude could become like those children. That's our hope," Jude's parent's said.

Coincidentally, low plasmalogen levels are also seen with Alzheimer’s disease.

"It's an amazing situation we're dealing with one of the rarest diseases in the world, rcdp is 1 in 100,000 and probably the most prevalent disease of our time which is dementia and aging -- at the same time. That doesn't happen very often."

Some hoping the connection brings light to this rare disorder.

"With those two similarities it definitely puts us more on the map I think."

If the trial is successful, it won't fix everything.

"But what is fixable and what the hope is we're going to raise the plasmalogens in their lungs and liver and brain and lungs is what many of our kids die from."

It's an emotional weekend for Patti and Brian Davidson. They're here in memory of their rhizo kid, Reece, who passed away this spring.

"She was just a happy child and she just had such a contagious smile and laugh and she never gave up until the very end when she was just tired," said Patti Davidson.

For Jude's parents this strikes a chord. Knowing their child could be next and knowing the pain families like the Davidson’s are coping with.

"They are walking all of our greatest fears and I can't imagine that but they came with such grace and they still have so much hope in their eyes for our children," Jude's parent's said.