News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

During National Sickle Cell Awareness Month, the American Red Cross is asking people to consider making a blood donation to benefit sickle cell disease patients — like Marqus Valentine. After all, such donations are crucial for Valentine, who was told by physicians, just before turning 30, that he…

Children with sickle cell disease (SCD) who arrive at an emergency department with vaso-occlusive episodes (VOEs) are more likely to have a longer delay before treatment, receive less narcotics than recommended, and to be admitted to the hospital, compared with SCD patients who present to a specialty day hospital, a…

A community town hall is set for Detroit to raise awareness about sickle cell disease, with a discussion about current and future therapies, as part of the Sideline Sickle Cell campaign by Emmaus Life Sciences, together with the Sickle Cell Disease Association of America Michigan Chapter. The…

A medication used to treat high blood pressure, called valsartan, may help decrease the stickiness of red blood cells and reduce the risk of vaso-occlusive crisis in people with sickle cell disease (SCD), a research study suggests. The study, “Valsartan impedes epinephrine-induced ICAM-4 activation on normal, sickle cell…

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,…