Treatment outcomes for people with sickle cell disease (SCD) have improved significantly in the past few decades. Today, over 90 percent of children who are born in Western countries with the genetic condition will live to adulthood. This is a remarkable achievement, as SCD was once synonymous with childhood mortality…
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…
A published case study reports that patients with mildly symptomatic sickle cell disease (SCD) can exceed the U.S. median life expectancy of 47 years for patients with the disease if it is managed properly. The report published in Blood, the Journal of the American Society of…
Researchers at various institutions from the African country of Cameroon recently found a link between oxidative stress and sickle cell disease (SCD). The study, “Oxidative profile of sickle cell patients in a Cameroonian urban hospital,” was published in…
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…
High levels of the protein albumin, a condition known as albuminuria and highly prevalent in sickle cell disease (SCD) patients, has now been linked to dysfunctions in cells that line the blood vessels (known as endothelial cells) in SCD patients. The study, “Albuminuria Is Associated with Endothelial Dysfunction and Elevated…
A Phase 2 clinical trial, aiming to assess the effects of hydroxyurea-based therapy at the maximum tolerated dose to prevent stroke in children with sickle cell anemia (SCA), began recently, and the outcomes are expected to highly impact children worldwide, particularly those living in developing countries where the disease is…
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