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December 5, 2019December 5, 2019

Adakveo Being Tested in Series of Trials, Including at Higher Dose and in Children

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A Phase 3 trial is testing a higher dose of Adakveo (crizanlizumab), the first approved targeted treatment for sickle cell disease, to explore if that dose might be more ... Read more

December 3, 2019December 3, 2019

HHS Secretary Alex Azar Touts White House Efforts to Fight Sickle Cell Disease

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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 ... Read more

November 27, 2019November 27, 2019

Oxbryta to Enter ‘Confirmatory’ Trial in Sickle Cell Children in Coming Weeks, GBT Says

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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 ... Read more

November 26, 2019November 27, 2019

FDA Approves Oxbryta, 1st Treatment Targeting Root Cause of Sickle Cell

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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 approval by the ... Read more

November 26, 2019November 26, 2019

Hemoglobin-rich Droplets Within Blood Cells May Be Source of Sickle Shape, Study Says

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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 ... Read more

November 22, 2019December 5, 2019

Adakveo ‘Important’ and Safe New Treatment for VOC Crises in Sickle Cell, SUSTAIN Researcher Says

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Adakveo (crizanlizumab) is an “important addition” to the care of sickle cell disease, a safe and effective therapy that works against painful vaso-occlusive crises (VOCs) in SCD patients and ... Read more

November 20, 2019November 20, 2019

CTX001 Found to be Safe and Effective in First Tested Patient with Severe Sickle Cell Disease, Preliminary Data Suggest

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CTX001 safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises in the first severe sickle cell disease (SCD) patient receiving the therapy, according to preliminary data from a ... Read more

November 19, 2019November 19, 2019

Universal Screening Tool for Socioeconomic Needs May Improve Care of Children with SCD, Study Suggests

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Universal screening to identify socioeconomic needs of children and families affected by sickle cell disease can help to address their specific difficulties, and ultimately improve the healthcare of those ... Read more

November 18, 2019November 19, 2019

FDA Approves Novartis’ Adakveo, 1st Treatment for Pain Crises in Teens and Adults with SCD

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A first targeted treatment for sickle cell disease (SCD) — Adakveo (crizanlizumab), by Novartis, that works to lower the frequency of the painful vaso-occlusive crises (VOCs) common in this ... Read more

November 14, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more