Behind the Mystery: Sickle Cell Disease
December 9, 2020
In the United States, sickle cell disease affects an estimated 100,000 people. Patients with severe sickle cell have a very poor quality of life, with frequent pain episodes and trips to the hospital. There is no universal cure for the disease, and patients typically only live into their 40s.
The disease slowly erodes their organs, so patients can develop chronic organ damage and then the loss of function of different organs. There are currently no universal treatment options for the disease.
However, research from Dr. Malik’s lab at Cincinnati Children’s is providing hope. She has developed a one-time, potentially curative investigational gene therapy called ARU-1801, which is currently in a clinical trial. Plus, at 47 years old, Carla Howard has already outlived the average life expectancy. She has made a decision to do everything she can to make a difference for the next generation.
Primary HLH is a rare, rapidly progressive, and fatal disorder with a median survival of less than 2 months.
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