Parents of Children with Hunter’s Syndrome Urge FDA to Restart Clinical Trial

It is a gut-wrenching plea for help.

“Please, please give them the right to live,” said Deena Leider, the boys’ mother.

Deena and Jeff Leider are desperate. They are desperate to save both of their boys from the grim, horrific reality of a little known and ultra-rare disease called Hunter syndrome. There are only six cases in New Jersey, and two of them in this household alone.

Hunter syndrome is a genetic disease where an enzyme the body needs is either missing or does not work.

“And it pretty much results in damaging all their organs and their brain,” Deena Leider said.

Most children lose their battle between the ages of 10 to 15. Jason is 7 while Justin is 4.

They are wonderful, playful brothers who are racing against the clock. With each passing month, Hunters takes more of their ability to live, but the FDA is testing a drug that could help them.

Case Hogan, 6, is proof of that. We talked via Skype with his mom, Melissa, from Tennessee. Before the clinical trial, Case was a danger to himself and losing IQ points monthly. Now with the drug, he is thriving.

“If we are out in public, say at a park, most people wouldn’t know there was anything wrong with him at all,” she said.

It’s been a huge difference, but the trial is on hold while the FDA works out some kinks in the way the drug is delivered. With every passing day, Jason is getting worse. So Jeff and Deena went to Congressman Bill Pascrell.

“You want to wrap your arms around them. And I do. so I am very proud of both of them. And we are going to help their son, come hell or high water,” Rep. Pascrell said.

He has written a letter urging the FDA to start up the trial again. Still waiting to hear back. That is all the Leider’s are asking for, a fighting chance.

“I can’t get the drug. And this little boy is 5 feet from Jason and he’s getting the drug and he’s living. My son is dying,” said Jeff Leider, the boys’ father.

“If there is something out there, give us some hope. Give us some chance. That’s all we ask,” Deena Leider said.

The Leiders have created a foundation called Let Them Be Little. It raises awareness of Hunter syndrome and helps parents of special needs kids. For the Leiders, the clock is ticking. They are trying to save their sons’ lives by getting them the drug. All the while, they are thinking of their third child. She is a little girl who does not have Hunters, but she could be carrying the disease with the risk of passing it to her children someday. For the Leiders, this nightmare never ends.

For more information, please visit the Leider’s website.