News

Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications that may be effective, with minimal safety issues, in treating sickle cell disease (SCD). Their findings were reported in the Journal of Clinical Medicine, in the study, “Large-Scale Drug Screen Identifies…

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. Baby’s First Test is a program of Expecting Health, an organization focused on pregnancy and newborn health. The…

Genetic analyses of African patients identified several genes and biological pathways that are associated with disease severity and longevity, and that might serve as new therapeutic targets in sickle cell disease (SCD). The study, “Genetic modifiers of long‐term survival in sickle cell anemia,” was published in…

Microbes in the gut play a key role in inducing vaso-occlusive crises (VOCs) in response to stress, a study in mice suggests. Stress is known to trigger VOCs in people with sickle cell disease (SCD) and inflammation in those with cardiovascular disease, but in ways not fully understood.

The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) who develop COVID-19, will be expanding to include information on children and adults in Africa. The expansion was funded by a $100,000 grant from the Doris Duke Charitable Foundation (DDCF) awarded to…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

September is National Sickle Cell Awareness Month, set aside annually to bring attention to the blood disorder and the needs of the community. From securing local government proclamations and hosting awareness events, to distributing information and lighting public spaces red, supporters have begun observing the monthlong event. The theme…

The European Commission has granted orphan drug designation to IMR-687, Imara’s potential new therapy for sickle cell disease (SCD). In the European Union, this designation is given to investigative therapies for rare diseases — those that affect fewer than five in 10,000 people — to…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential cell therapy for the treatment of sickle cell disease (SCD). This designation is given to therapies that aim to treat serious or life-threatening disorders that affect fewer than 200,000 people…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…