CHICAGO, Illinois -- A toddler from California is being treated in Chicago for a genetic disorder that can be deadly.
Marian McGlocklin, just 19 months old, is battling a disease that is often called "childhood Alzheimer's."
Years ago, there was nothing that could be done to treat the disease. But now, a drug in clinical trials is offering hope to families around the world.
Rush University Medical Center is doing compassionate treatment for children not in the trials, including for families visiting from Texas, Canada and Costa Rica, as well as the youngest patient, from California.
Marian is just about with her peers with her language and cognition, but she isn't walking yet. The reason is because of a rare genetic disease that affects the brain called Niemann Pick Type C, which some call "childhood Alzheimer's."
It is a fatal disease, but an experimental drug is showing promise in Chicago. So every two weeks, Marian is brought to Chicago for the treatment. Monday was her second visit to Rush University Medical Center.
"I feel really frightened," her mother, Sara McGlocklin said. "But extremely, extremely grateful and hopeful."
A fundraising campaign in Los Angeles is helping her family with travel and medical expenses. Her older sister held a lemonade stand, and there is other support such as the family friend who traveled with Marian and her mom.
"I couldn't imagine not being here," Christine Allen said. "You need all hands on deck."
Dr. Elizabeth Berry-Kravis, a Rush University Medical Center pediatric neurologist, said Marian is the youngest patient treated with VTS-270. She said the drug has slowed the disease's progression in other patients.
"Either we can keep them from progressing long enough that we discover other new things that we can also use, or we keep them from progressing for a very long time," she said.
Her mother is just hopeful for the best possible outcome.
"I hope that she is going to live a long and healthy and happy life," Sara McGlocklin said. "I just want her to be happy. And I want my girls to have each other as sisters. They love each other so much."
In addition to funds raised for the family, colleagues of Sara McGlocklin have donated their vacation days to allow McGlocklin more time to spend with Marian and travel to Chicago appointments.
Eventually Marian's doctor expects a facility in California will be able to administer the drug which is done with a spinal tap.
California toddler being treated for 'childhood Alzheimer's'
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