Margarida Maia, PhD,  science writer—

The U.S. Food and Drug Administration has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives like…

Preschool-aged children with sickle cell disease (SCD) living in so-called food deserts, or areas where access to healthy food is limited, and those without reliable transportation are at a higher risk of experiencing health issues than other youngsters with SCD, even when they receive good medical care, a study…

The number of stroke cases among adults and children with sickle cell disease (SCD) continues to increase in at least one U.S. state, indicating a need for greater adherence to standard-of-care blood transfusions and more attention paid to factors that can be controlled to reduce the risk of…

Using lactated Ringer’s solution instead of normal saline to replace lost fluids in adults admitted to the hospital for vaso-occlusive crises, that is, pain episodes caused by sickle cell disease (SCD), allows for shorter stays, fewer readmissions, and a reduced need for opioids, a study finds.  Dehydrated…

Low-intensity stem cell transplants, which use milder conditioning regimens, appear not to damage the lungs and may even improve lung function in adults with sickle cell disease (SCD), according to a new study led by scientists from the National Institutes of Health (NIH). “By using a low-intensity blood…

A second clinical study to test how well BioLineRx’s motixafortide helps mobilize hematopoietic stem cells for use in gene therapies in sickle cell disease (SCD) is expected to begin enrolling in the next few months. The planned Phase 1 study, called SCDSTEMM (NCT06442761), will run as a…

Explosive synchronization in the brain, wherein a small change in the brain network causes it to sync up and undergo a sudden shift in behavior, could be behind frequent pain crises in sickle cell disease, according to a study that may offer a way to better predict the disease’s…

A bone marrow transplant approach that doesn’t use high-dose chemotherapy to prevent an immune response against a donor’s stem cells showed high rates of event-free and overall survival among sickle cell disease (SCD) patients treated in a Phase 2 clinical trial. The approach, which also expands the eligibility of…

Oxbryta (voxelotor), an oral medication approved to treat sickle cell disease (SCD), prevented children’s red blood cells from stiffening into a characteristic sickle-like shape and breaking down, possibly helping more oxygen to get into tissues in the body. That’s according to a study reporting data on 10 children…

While children with sickle cell anemia — the most common form of sickle cell disease — are vulnerable to serious infections and stroke, many don’t receive the antibiotics or annual ultrasounds that national guidelines suggest could keep them healthier, a study found. Only about 1 in 5 children took…