Medical Mysteries: A tiny baby who didn’t grow

Katherine Frey/THE WASHINGTON POST - Morgan McElhinney, who weighs 45 pounds at age 9, relaxes at home in Brunswick, Md., with her parents, Lisa Simonson McElhinney and Brad McElhinney.

Soon afterward, Morgan’s parents took her to a neurologist at Johns Hopkins Hospital. He ruled out cerebral palsy, a birth injury or a structural problem with Morgan’s brain. His diagnosis: “global developmental delay,” which seemed more description than explanation.

“I was just very frustrated, but at least I knew the hospital hadn’t done anything wrong,” said McElhinney, who worried that something had happened during delivery to cause Morgan’s problems or that she somehow was responsible. “But when you don’t know what the diagnosis is, you don’t know what to expect” or how best to treat it.

(Katherine Frey/THE WASHINGTON POST) - Morgan McElhinney uses a device similiar to an iPad to communicate. The girl does not speak, but she has an excellent memory and appears to understand a lot, says her mother, who helped found a group to raise awareness of her rare disorder.

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A second visit to the neurologist when Morgan was 15 months old and had just begun to sit unsupported — nearly a year after most babies do — was similarly unrevealing. Doctors ruled out autism and several rare chromosomal maladies, but they couldn’t pinpoint the underlying problem.

‘Am I ever going to know?’

“We basically just kept trying to get her development going,” through the early intervention program, said McElhinney. As long as Morgan was making slow progress that seemed the best thing to do, especially as a protracted family crisis demanded considerable attention and resources.

“It’s hard,” she said. “I work a full-time job and have three other kids.” She and her husband decided to devote their energies to making sure Morgan got the help doctors said she needed and to put aside their search for a diagnosis.

But in the fall of 2007, when Morgan was 5, a local neurologist who had been seeing her suggested that the family consult specialists at the Kennedy Krieger Institute in Baltimore, which treats children with developmental disorders.

In January 2008, McElhinney took Morgan and her voluminous medical records to Kennedy Krieger. “I really thought, ‘Oh, my God, am I ever going to know what this is?’ ” she recalls.

After taking a detailed family history and performing some tests, the developmental pediatrician said something McElhinney had waited five years to hear: “We think we know what this is,” she remembers being told. A definitive diagnosis would require confirmation by geneticists at Hopkins.

Morgan met many of the criteria for Cohen syndrome, a very rare genetic disorder first described in 1973 by Canadian physician Michael Cohen. To date, only about 1,000 cases of the disorder have been identified worldwide, although doctors agree that many more have never been diagnosed.

Cohen syndrome results from the mutation of the VPS13B gene, which may be involved in the sorting of proteins in cells, according to the Genetics Home Reference Web site. Most people with the syndrome, which ranges in severity, experience growth problems, delayed development, intellectual disabilities including mental retardation and low muscle tone, and they have smaller than normal heads at birth. Heart defects and retinal problems leading to blindness can also occur. Most patients inherit two defective copies of the gene, one from each parent .

There is no cure for the syndrome, which is overrepresented among certain groups where marriage to outsiders has been uncommon, allowing rare genetic flaws to be perpetuated. These groups include the Amish, people of Finnish ancestry, a nomadic Irish group known as Irish Travellers, and residents of an isolated Greek island.

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