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California’s legislature has committed $15 million toward sweeping enhancement of adult sickle cell disease (SCD) care in a state with historically underserved patients. The initiative is intended to improve care largely through creation of a statewide network of regional adult SCD clinics modeled after the newly opened clinic at the Martin Luther King, Jr. Outpatient Center in Los Angeles, an area where nearly 2,000 patients reside. Called the Jeffrey Smith Sickle Cell Adult Center, the comprehensive...


By 2029, Americans with sickle cell disease (SCD) could be living 10 years longer than they do today, predicts U.S. Health and Human Services Secretary Alex Azar. That’s one of the Trump administration’s long-term goals, Azar — the nation’s top health official — told more than 900 delegates attending the 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit in Washington, D.C. U.S. Health & Human Services Secretary Alex Azar addresses the 2019 NORD Summit...


Oxbryta (voxelotor), the first treatment for sickle cell disease that addresses a root cause, will continue to be tested in a post-approval confirmatory study opening soon in children as young as age 2, according to its developer, Global Blood Therapeutics (GBT). The new study, called HOPE-KIDS 2, is expected to “be initiated” by year’s end, Steven Immergut, a senior vice president and head of corporate communications at GBT, said in an email response to questions. Its design was...


Droplets packed with hemoglobin may briefly form tiny clusters inside red blood cells that ultimately deform their shape, and cause the pain crises that mark sickle cell disease (SCD), a study reports. The research, “A mechanism for reversible mesoscopic aggregation in liquid solutions,” appeared in the journal Nature Communications. In people with SCD, hemoglobin (the protein in red blood cells that carries oxygen) can form rigid protein strands in the blood cells that alter their shape. These...


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