From walks to blood drives, a host of activities and efforts are marking this year’s National Sickle Cell Awareness Month, observed annually each September. For its part, Nationwide Children’s Hospital, which has a Comprehensive Sickle Cell and Thalassemia Program, is reminding people of the importance of being tested…
The U.S. Food and Drug Administration (FDA) is giving priority review, as requested, to Global Blood Therapeutics‘ application to approve voxelotor as a treatment for sickle cell disease (SCD). Voxelotor, formerly known as GBT440, is as an oral and once daily potential sickle cell therapy. It works by…
Taking place during National Sickle Cell Awareness Month, the 8th Annual Sickle Cell Disease (SCD) Therapeutics Conference will focus on the latest medical advances and developing treatment trends. To be held Sept. 10 in Washington, D.C., the conference will be hosted by the Sickle Cell Disease Association…
African Americans who inherit one sickle cell gene and one normal gene — a condition known as sickle cell trait — are not at greater risk of cognitive decline or dementia. However, such genetic composition may influence other risk factors for cognitive impairment, researchers report. The study with that finding,…
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
A new computer model is able to simulate how red blood cells become misshapen in patients with sickle cell disease. The model may be useful in identifying which medicines could be beneficial for preventing the development of sickled cells. The study about that research, “Quantitative prediction of erythrocyte sickling for…
The former professional rugby player Ade Adebisi, a sickle cell disease patient and advocate, has been named brand ambassador for Sickle Scan, BioMedomics’ rapid disease diagnostic tool. Currently vice chairman and general manager of the Nigeria Rugby League Association, Adebisi will represent what’s touted as the world’s first rapid…
ReachBio Research Labs has created a drug screening platform to discover therapeutic compounds that trigger the production of fetal hemoglobin (HbF) to treat sickle cell disease (SCD) and other blood disorders. SCD belongs to a group of inherited disorders known as hemoglobinopathies, primarily caused by mutations in…
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…
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