Sunday, December 07, 2025

National push to open access to sickle cell gene therapies

Breakthroughs in cell and gene therapy have opened the door to life-changing treatment for people with sickle cell disease. Yet few patients know these treatments exist, and even fewer can access them due to the seven-figure price tag.

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... Across the country, fewer than 100 people have undergone gene therapy for the disease, said Dr. Lakshmanan Krishnamurti, chief of pediatric hematology, oncology, and bone marrow transplant at Yale New Haven Children's Hospital.

In an effort to expand access, Connecticut has joined 33 states, Washington, D.C., and Puerto Rico in a first-of-its-kind federal program to make the therapy available through Medicaid. Together, these states account for 84% of Medicaid recipients living with sickle cell disease.

An estimated 100,000 people in the United States have sickle cell disease, a condition that primarily affects people of color, according to the Centers for Disease Control and Prevention. More than 90% of those affected are Black, and an estimated 3% to 9% are Latino. ...


#1 | Posted by LampLighter at 2025-12-07 01:15 AM

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