News

The U.S. Food and Drug Administration recently accepted an investigational new drug (IND) application submitted by Bioverativ and Sangamo Therapeutics for BIVV003, a gene-edited cell therapy candidate for the treatment of sickle cell disease. BIVV003 uses a nonviral approach that uses zinc finger nuclease (ZFN) gene-editing technology to…

Physicians who use stigmatizing language in their patients’ medical records could be affecting the care they receive for years to come, according to a new Johns Hopkins study. Titled “Do Words Matter? Stigmatizing Language and the Transmission of Bias in the Medical Record,” and published in the Journal…

Three programs focused on sickle cell disease (SCD) planned by St. Jude Children’s Research Hospital will benefit from a $1 million Legacy Grant awarded by The Links Foundation. The foundation, the philanthropic arm of The Links — one of the United States’ oldest and largest African-American women’s volunteer service…

Under a new treatment protocol, seven adults with sickle cell anemia were treated — and reportedly cured — using stem cells from donors previously seen as incompatible. The study reporting these treatments, “Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease,” was…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

It’s been nearly half a century since the end of Alabama’s Tuskegee experiment, the infamous 40-year study in which the U.S. government intentionally gave 399 syphilis-infected black men useless placebos — like aspirin and mineral supplements — instead of penicillin, which could have cured them. Yet suspicions still linger, complicating today’s…

It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact, it rarely happens. But this year is different. Thailand’s Prince Mahidol Award Foundation has chosen the National Human Genome Research Institute — a division of the National Institutes of Health (NIH)…

In-person education of caregivers increases their knowledge of sickle cell trait (SCT), according to researchers. But new strategies are needed to make sure caregivers don’t forget what they learned. The study, “Sickle cell trait knowledge and health literacy in caregivers who receive in‐person sickle cell trait education,” appeared in…

The amount of oxygen the blood loses when traveling through the brain could help identify children with sickle cell disease who are at risk of having a stroke, a study reports. Scientists call the measurement the oxygen extraction fraction because it refers to the percentage of oxygen that the brain…

Novo Nordisk has obtained worldwide rights to EpiDestiny’s sickle cell disease treatment EPI01. The disease-modifying therapy, consisting of decitabine and tetrahydrouridine, is aimed at  helping patients increase fetal hemoglobin levels to replace the hemoglobin that’s defective in the disease. The idea is to prevent red blood cells from becoming sickle-shaped,…