La Jolla’s investigational therapy LJPC-401 (synthetic human hepcidin) to treat sickle cell disease has been recommended for orphan drug designation through a positive opinion issued this week by the European Medicines Agency‘s (EMA) Committee for Orphan Medicinal Products (COMP). “We are encouraged by the positive feedback and continued support of…
National and local policies have led to large reductions in lead poisoning cases overall in the U.S., but severe cases are still common among children. Recently, researchers at the New York City Department of Health and Mental Hygiene (DOHMH) investigated sources of exposure, risk factors, and outcomes for children with severe lead poisoning. Sickle cell…
For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells. In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation…
Mast Therpeutics has announced that the clinical development of Vepoloxamer (MST-188) is likely to be terminated. Results from a Phase 3 clinical trial evaluating the investigational drug in patients with sickle cell disease (SCD) failed to meet its primary goal. Brian M. Culley, Mast Therapeutics’ chief executive…
In his keynote speech at a University of Miami symposium recently, Robert M. Califf, the U.S. Food and Drug Administration (FDA) commissioner, marveled about how scientists soon may be able to simply replace disease-causing genes with healthy ones. Gene editing may transform the lives of patients affected with several diseases, including…
Engineers and computer scientists from the University of Washington (UW) have developed what they are calling a HemaApp, designed to detect hemoglobin concentration using simply a smartphone camera and a little extra lighting — rather than needles or an expensive, specialized machine. Measuring hemoglobin, a protein found in red blood cells,…
Sickle cell disease (SCD) is rampant in sub-Saharan Africa, a region that accounts for about two-thirds of all SCD births worldwide, and among the disease’s complications can be an imbalance in elements such as iron, an essential trace element for good health. Researchers compared key proteins associated with iron metabolism between children with SCD and…
Researchers believe that activating a specific oxidative stress regulator with sulforaphane (SFN), a chemical compound found in vegetables such as broccoli and brussels sprouts, might have a therapeutic benefit in patients with sickle cell disease (SCD). They tested their hypothesis in a Phase 1 study that evaluated broccoli sprout homogenate (BSH), which naturally contains SFN,…
The Embrace Kids Foundation has committed $70,000 to support the expansion of Rutgers Robert Wood Johnson Medical School (RWJMS) Comprehensive Sickle Cell Center at Rutgers Cancer Institute of New Jersey (CINJ) to include a pediatric sickle cell and hemoglobinopathies nurse navigator position. The main focus of the new position is…
The American Red Cross and Sports Clips Haircuts have partnered to increase blood and platelet donations across the U.S., offering a coupon for a free haircut to all those who donate during September. Blood donations can be life-saving for people with chronic conditions like sickle cell disease (SCD). “The Red Cross and Sport…
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