Posted by: Hemalatha Rangarajan, MD on Jul 28, 2025

Something exciting happened in December 2023, when the U.S. Food and Drug Administration approved a gene therapy for the treatment of sickle cell disease in adult and pediatric patients 12 years and older who require regular red blood cell transfusions. Nationwide Children’s Hospital is one of a handful of facilities in the United States that is certified to have the advanced facilities and specially trained staff which are needed for the administration of the therapy.

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Down Syndrome Regression Disorder: Not One-Size-Fits-All

For as much as we think we know about Down syndrome, there is still more and more that we are learning. In fact, we are sometimes explaining things to families at the same time we are learning them.

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Turner Syndrome: Unique as a Butterfly

Turner Syndrome is a genetic condition, a difference in the number or type of sex chromosomes, in those assigned female at birth.