CHARLOTTE, N.C. — Lori Collins, and her 10-year-old daughter Katelyn are fighting for their lives each day with a rare disease called chronic mucocutaneous candidiasis.
The disease permits them to fight off many infections that most people can. Collins’ father and sister died from the illness.
About three years ago, Katelyn developed complications connected to the disease, which damaged her liver and red blood cells, and caused extreme side effects.
A bone marrow transplant could have helped, but doctors at Levine Children's Hospital said it would have been risky.
Doctors instead ordered a new drug that’s only been given to a few patients in the world.
Katelyn has been taking the medication since May, and it’s been working.
“Katelyn is very strong, and I try to be,” Collins said. “I think we try to put up a front because we don’t want people to worry.”
The medication was approved in 2011 by the Food and Drug Administration, and it costs the family $16,000 a month.
“It’s a lot, but it's saving her life, so it’s worth every penny,” Collins said.
Katelyn said she’s been able to return to everyday activities.
“I’ve been able to do gymnastics,” Katelyn said. “In fact, I have gymnastics tonight.”
The family's doctor, Dr. Niraj Patel, believes that same medication could now help others.
“There may be more incidences where we use that medication to treat patients similar to Katelyn,” said Patel, the Chief of Pediatric Infectious Disease and Immunology at Levine Children’s Hospital.
Thursday is “Rare Disease Day,” which recognized people that affects less than 200,000 people.
Channel 9 learned that Levine Children's Hospital is conducting more cutting-edge trials this year that are aimed at helping more children fighting rare diseases.
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