Local 9-year-old battling rare disease has new ray of hope

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HARTFORD -- One Connecticut family has renewed hope after Italian scientists claim they have found a cure for a rare disease.

Hunter Pageau, 9, made an impression at the Capitol on Monday when he eloquently spoke to reporters about his ordeal.

With a smile ear to ear and manners that any parent would be proud of, Hunter is nothing short of inspirational. On this day he has every reason to grin, as the New Haven kid recently learned there may be a cure for the disease that's left him wheelchair-bound and hooked up to a ventilator. "It feels great to be here today, thank you,” he told reporters.

Hunter suffers from "SMARD," or Spinal Muscular Atrophy with Respiratory Distress. It's incredibly rare. Hunter is only one of ten people in the U.S. and 80 worldwide who has the disorder. "I was so amazed and so excited when I first heard about that,” said Hunter of the possible cure.

Researchers published their new research last week saying they've found a specific type of gene therapy that treats, and may even reverse the effects of SMARD. They can now move forward with human clinical trials.

"We received this news a few days ago,” said his mother, Sharon Pageau. “Prior to this news there had been no clinical proven path to a cure. The door has been formally thrown wide open for a cure for Hunter's extremely rare disease.”

Sharon says it may mean Hunter could stand, walk and maybe not need to be hooked up to a ventilator. This comes as the family rallies for legislation at the Capitol to create "SMARD Awareness Day." The bill passed out of committee.

"It shows you what happens when you bring awareness to the issue,” said Sen. Len Fasano (R-North Haven). “You get people involved, people understand what Hunter's been through, there's an effort to work to resolve the issue."

And it also proves what Hunter has said all along.

"My message for everyone is hope, love, and never, ever, give up,” said Hunter. “Whatever you do, never give up."

Another piece of good news is legislation currently in the Public Health Committee. Sen. Fasano says the bill would, under certain circumstances, allow a person to partake in a clinical trial for a drug before it is approved by the FDA. This could also help Hunter in the future.

Sharon says their family's next goal is to get Hunter to Italy so he can partake in clinical trials.

To donate so that Hunter can travel to Italy for the trials, click here, and for more information on Hunter's story you can visit HOPE for Hunter Pageau on Facebook.