And when the transplant is done before symptoms appear, infants show continued gains in developmental skills.

Hunter's Hope -- established by Jill and Jim Kelly in 1997 after their son, Hunter, was diagnosed with Krabbe's -- helped fund the study. The results were published in this week's New England Journal of Medicine.

"This research validates what we've known all along, from meeting with the children who have had the transplants and with the doctors and researchers from the study," said Micki Gartzke, the foundation's education director, whose daughter, LeA, died of Krabbe's disease in 1998, at age 2.

Results Buoy Hunter's Hope

The study followed 11 newborns who underwent cord-blood transplants -- transfusions of blood from a newborn's umbilical cord -- before they showed any symptoms. Another 14 infants were given transplants after they developed symptoms.

The children were studied for four months to six years. After that time, all 11 who received transplants before showing symptoms had survived, compared with six of the 14 who had transplants after symptoms appeared.

Krabbe's disease, an inherited, degenerative disorder, eats away at the nervous system, often taking an infant's life within one or two years. Younger children often are tested only because an older sibling had been afflicted.

"The bottom line is that it works on the children who are transplanted before their symptoms show up," Gartzke said. "And on the surviving] children who were transplanted after their symptoms, it stopped the progress of the disease, but they didn't see any significant gains."

The results buoy Hunter's Hope officials for several reasons:

The foundation provided money and data for the study, plus support for families that go to Duke for the transplants.

The study will make doctors aware that such a transplant represents sound medical treatment.

Screening All Newborns

"When Hunter was diagnosed, we were told that there wasn't any treatment," said his grandmother, Jacque Waggoner. "That treatment was happening, but it was considered experimental."

The study will help Hunter's Hope push one of its top priorities: screening all newborns for the disease.

"It's going to help the medical field understand the importance of newborn screening," said Waggoner, chairwoman of the foundation. "This study shows very clearly that you have to treat the child before the symptoms present themselves."

David Margolis, director of the bone marrow transplant program at Children's Hospital of Wisconsin and associate professor of pediatrics at the Medical College, said the transplant is a very straight forward process and is something that could easily be done at his hospital.

But, he said, transplants are not slam-dunks -- they are risky procedures.

"There is the sad, realistic possibility that someday soon, a child will die from a transplant-related complication."

But the big question is how many will survive and how many years will the treatment be effective?

"Our hope is that it will be a permanent fix," Kurtzberg said.