News

The Green Park Collaborative (GPC) is working with the Sickle Cell Disease Association of America (SCDAA) to develop a consensus of desired outcomes to be used in clinical studies evaluating new treatments for sickle cell disease (SCD). The project, named coreSCD, aims to deliver a list of recommendations on…

Consumers may know Pfizer as the company that manufactures Viagra, but the New York-based pharma giant is investing millions in developing potential treatments and gene therapies for rare disorders ranging from sickle cell disease and hemophilia to ALS and Duchenne muscular dystrophy. Michael Wajnrajch, MD, is senior medical director…

Children and adolescents with sickle cell anemia (SCA) showed low adherence to treatment with hydroxyurea, according to a multi-state study in the U.S. The research, “Hydroxyurea use among children with sickle cell anemia,” was published in the journal Pediatric Blood & Cancer. Hydroxyurea is…

Alterations in the lungs’ network of blood vessels, caused by sickle cell disease (SCD), reflect disease burden and should be carefully monitored over time, a recent study says. The study with that finding, “Clinical relevance of pulmonary vasculature involvement in sickle cell disease,” was published in the…

Development of problem-solving skills may help adolescents with sickle cell disease prepare for the transition to adulthood and also improve their ability to self-manage their health. Researchers at the Boston University Medical Center conducted a study to gain better insight on the perspectives of sickle cell disease (SCD)…

The success rate of bone marrow transplants for patients with sickle cell disease or beta thalassemia, from only “half-matched” donors, increased by doubling the radiation delivered to patients’ bodies before the transplants, a John Hopkins University study shows. The study, “Effect of increased dose of total body irradiation on…

Oral morphine treatment is a valid therapy option for patients with sickle cell disease (SCD) who are undergoing a vaso-occlusive crisis (VOC), researchers suggest. It was found to significantly reduce the rate of hospitalizations and the number of intravenous treatments patients must undergo to control these crisis. The…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…

A Republic of Congo delegation of health and higher education representatives recently met with Howard University officials, aiming to ultimately advance research into sickle cell disease (SCD) — a particularly pervasive disorder in Africa. Led by Congo Minister of Health and Population Jacqueline Lydia Mikolo and Minister of Higher…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…