News

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

November 14, 2019November 14, 2019

Modified CRISPR/Cas9 May Open Door to New Therapies for SCD and HIV

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Scientists have found a way to increase the effectiveness of the gene-editing tool CRISPR/Cas9 that one day may be used to treat people with sickle cell disease (SCD) and ... Read more

November 12, 2019November 12, 2019

Gene-Editing Tool in Ongoing Trial of BIVV003 in Treating Severe Sickle Cell Supported by Early Study

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A technique called zinc finger nuclease (ZFN) gene editing technology can be used to modify immature red blood cells — called precursor cells — to boost the production of ... Read more

November 7, 2019November 7, 2019

Delta-aminolevulinate Molecule May Be Potential New Therapy for Sickle Cell Anemia, Study Suggests

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Treatment with delta-aminolevulinate (ALA), a precursor of heme — the part of hemoglobin that carries oxygen — could be a future therapy option for people with sickle cell anemia ... Read more

November 5, 2019November 5, 2019

Inflammation in Airways of Sickle Cell Children Distinct from Asthma Alone, Pilot Study Suggests

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White blood cells, especially monocytes, may underlie the breathing difficulties affecting children with sickle cell disease (SCD), a small pilot study suggests. Asthma is estimated to impact from 17% ... Read more

October 31, 2019October 31, 2019

NIH and Gates Foundation Each Invests $100M to Boost Development of Affordable Gene Therapies for Sickle Cell Disease, HIV

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The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable ... Read more

October 29, 2019October 29, 2019

Enhanced Parenting Skills May Help Improve Cognitive Function in SCD Children, Study Suggests

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Enhanced parenting skills, including attentive listening and engaging in conversations, may help improve cognitive functioning in children with sickle cell disease (SCD), a study suggests. Parent stress was related ... Read more

October 24, 2019October 24, 2019

Caraphenol A May Facilitate Faster, More Effective Gene Therapies for Sickle Cell Anemia, Study Says

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Caraphenol A — a small molecule closely related to a natural compound called resveratrol, which is produced by red grapes — increased the efficacy of hematopoietic stem cell-based gene ... Read more