Emmaus Life Sciences announced that expects to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) next month for its lead investigational treatment for sickle cell disease. Emmaus’ lead candidate is an oral pharmaceutical grade L-glutamine treatment that demonstrated positive clinical results in…
News
The William E. Proudford Sickle Cell Fund in celebration of September’s National Sickle Cell Awareness Month is recognizing the accomplishments of Dr. Gary Gibbons, Director of the National Heart, Lung, and Blood Institute (NHLBI). “Under the leadership of Dr. Gibbons, NHLBI has demonstrated…
In sickle cell anemia (SCA), infection with the erythrovirus B19 (B19V) is known to cause transient aplastic crises, in which production of red blood cells (RBCs) in the bone marrow fails. In this study, researchers describe the clinical course and lab tests of two brothers with sickle cell disease who had the infection.
Researchers developed a mobile app to provide primary care physicians (PCPs) with tools — guidelines, algorithms, and a pathway — needed to communicate effectively with local specialists regarding sickle cell disease. The team from Duke University and the University of Pittsburgh developed the iOS-based ‘toolkit’ after finding that not all…
Pain is an important, and difficult to treat, feature of sickle cell anemia (SCA). Here, researchers compared two different types of mouse models of the disease to study pain and the effects of age and gender on pain development and behaviors. The study highlights how these animal models can increase understanding…
Possible Therapeutic Targets, Diagnostic Tools for SCA Heart Complications Identified by Researchers
Increasing evidence points to the development of cardiopulmonary complications in sickle cell anemia (SCA) that may lead to sudden death but cannot be solely attributed to the chronic anemia aspect of the disease. Here, researchers used SCA mouse models to study the physiological, molecular, and cardiac phenotype associated with SCA,…
Early diagnosis of infants affected by sickle cell disease (SCD) through neonatal screening and adequate follow-up are effective tools to decrease the mortality rate associated with the condition. Researchers in central India sought to establish a neonatal screening program and describe the clinical course and hematological presentation of babies with sickle cell.
Sickle cell trait, a form of sickle cell caused by one (rather than two) faulty alleles, has long been thought to raise a person’s risk of premature death from muscles breaking down under exertion, straining the kidneys. But a new study contradicts this assumption, finding no elevated risk of mortality…
Iron overload, a major concern in patients with sickle cell anemia (SCA), can lead to serious tissue damage in the skin and other important organs. Researchers at Mersin University investigated the clinical effectiveness of iron chelation therapy and its effect on the quality of life and satisfaction of SCA patients,…
Sickle cell anemia, although best characterized by chronic anemia, pain, and vascular  occlusion, is also associated with strokes and cognitive problems in children, and magnetic resonance imaging is key to proper monitoring and managing of the disease, researchers in China reported. Researchers at the Xuzhou Children’s Hospital highlighted the neurological complications of…
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