Girl with spinal muscular atrophy making progress in a clinical trial
When Elizabeth Lee Hallam was born on Sept. 29, 2003, she was a textbook example of the perfect baby. But by 8 months of age, she was battling a fatal genetic disease her family had never heard of. Despite the grave circumstances of her disease, Elizabeth is doing tremendoulsly well under a new therapy.
The research is in the initial stages, but if the treatment succeeds, Wang says it could give new hope not just to children afflicted with SMA but those with other genetic illnesses and other motor neuron diseases.
"This little girl is defying every rule, every possible odd," said Wang, director of the Neuromuscular Disorders Clinic at Packard Children's Hospital in Palo Alto, Calif. "She's going to turn 3, and she shouldn't even be here."
"The state of the field is much more encouraging than it has ever been in the past for SMA," said Dr. Kenneth Fischbeck, chief of the neurogenetic branch at the National Institute of Neurological Disorders and Stroke.
As it is, finances are tight. The airfare for Elizabeth and her mother is picked up by Mercy Medical Airlift, a charitable program, but Jeanna Huette must pay her own travel expenses, about $750 a month. That's over and above the regular medical costs, which are daunting enough for Christen Huette, who earns $6.75 an hour at the local Kmart and lives with her parents.
In August - two months after the trial started - all the hassles paid off.
Elizabeth was suddenly able to move her head. Next came her limbs with stronger muscle tone. Now, Elizabeth is chattering away, repeating her ABCs, counting to 10 and even managing a lip-curling, hip-swaying Elvis imitation.
The odds are still long, but the trips to California have given everyone something they didn't have before: hope. Family members slip easily into talking about Christmas, preschool and other future milestones.
"Whatever time we have has been a gift," said Christen Huette. "She's not giving up, so neither can we."
The research is in the initial stages, but if the treatment succeeds, Wang says it could give new hope not just to children afflicted with SMA but those with other genetic illnesses and other motor neuron diseases.
"This little girl is defying every rule, every possible odd," said Wang, director of the Neuromuscular Disorders Clinic at Packard Children's Hospital in Palo Alto, Calif. "She's going to turn 3, and she shouldn't even be here."
"The state of the field is much more encouraging than it has ever been in the past for SMA," said Dr. Kenneth Fischbeck, chief of the neurogenetic branch at the National Institute of Neurological Disorders and Stroke.
As it is, finances are tight. The airfare for Elizabeth and her mother is picked up by Mercy Medical Airlift, a charitable program, but Jeanna Huette must pay her own travel expenses, about $750 a month. That's over and above the regular medical costs, which are daunting enough for Christen Huette, who earns $6.75 an hour at the local Kmart and lives with her parents.
In August - two months after the trial started - all the hassles paid off.
Elizabeth was suddenly able to move her head. Next came her limbs with stronger muscle tone. Now, Elizabeth is chattering away, repeating her ABCs, counting to 10 and even managing a lip-curling, hip-swaying Elvis imitation.
The odds are still long, but the trips to California have given everyone something they didn't have before: hope. Family members slip easily into talking about Christmas, preschool and other future milestones.
"Whatever time we have has been a gift," said Christen Huette. "She's not giving up, so neither can we."
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