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…
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…
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.
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,…
Gamida Cell has announced that the first patient with sickle cell disease (SCD) has been transplanted with CordIn, the company’s experimental curative treatment for rare and chronic nonmalignant diseases with no other curative therapy options besides bone marrow transplant. The transplant, performed at UCSF Benioff Children’s Hospital Oakland, is part…
A comprehensive review of management approaches for childhood sickle cell disease (SCD), focused on neonatal screening implementation, common complications, and current standard treatment, drew a primary conclusion that improvements in the care of patients transitioning from pediatric to adult patients is needed to reduce young adult mortality and hospital costs. The review article, titled…
Get regular updates to your inbox.