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Written by By Jemela Williams I’ve been asked this question on more than one occasion when I mention the medications that I’m currently on for the management of my Sickle Cell Anemia. What is Endari? So I’m here to answer this question amongst others such as, what Endari is for, what it’s supposed to do, how do you use it, side effects and more. I’ll also include how my Sickle Cell SS has been affected...


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...


With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high costs and limitless potential for curing diseases — generated much discussion at the Washington, D.C., event, which was organized by the National Organization for Rare Disorders...


Ghana is working with Novartis to make hydroxyurea treatment available to people with sickle cell disease, and to open a clinical trial in the African country testing the company’s biologic SCD therapy Adakveo (crizanlizumab), recently approved in United States. The five-year, public-private partnership was formed in January among the Ministry of Health of Ghana, Ghana Health Service, the Sickle Cell Foundation of Ghana, and the pharmaceutical company. An estimated 15,000 babies are born with sickle cell disease in...


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...


Oxbryta (voxelotor), a first sickle cell treatment that works to stop the sickling and destruction of red blood cells that mark this disease, has been given accelerated approved by the U.S. Food and Drug Administration (FDA) for patients ages 12 and older. Specifically, Oxbryta — an oral, once-a-day tablet developed by Global Blood Therapeutics (GBT) — is designed to block sickle hemoglobin polymerization, the root cause of sickle cell disease. Mutant hemoglobin polymerization refers to the clumping and...


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