News

Advocacy groups across the U.S. are hosting events throughout September to mark Sickle Cell Awareness Month. The month has been recognized since the 1980s as a time to raise awareness about sickle cell disease (SCD), a genetic disorder that affects red blood cells. This year, advocacy groups including the…

Genetix Biotherapeutics is teaming up with the National Football League Alumni Association for a campaign to increase awareness of one-time genetic treatments for sickle cell disease (SCD). “Despite hundreds of patients now treated, the vast majority of eligible patients remain untreated, primarily due to lack of awareness that…

Common pain relievers, such as nonsteroidal anti-inflammatory drugs (NSAIDs) or acetaminophen, combined with standard opioid care, may provide meaningful relief for acute pain in people with sickle cell disease (SCD), according to a review study. Cognitive behavioral therapy, a type of talk therapy, also showed signs of efficacy against…

The first participant has now been dosed in a global clinical trial designed to confirm the benefits of mitapivat, an oral therapy under regulatory review in the U.S. for the treatment of sickle cell disease (SCD), its developer Agios Pharmaceuticals announced. The U.S. Food and Drug Administration (FDA)…

A team of experts from across the U.S. has created a new set of national guidelines on how best to provide cognitive and developmental care to people with sickle cell disease (SCD). The guidelines stress the importance of proactive assessments so that cognitive or developmental issues can be detected…

Agios Pharmaceuticals is discontinuing development of its oral therapy candidate, tebapivat, for people with sickle cell disease (SCD). The decision follows results from a small Phase 2 clinical trial (NCT06924970) showing that while tebapivat — an activator of the pyruvate kinase (PK) enzyme — reduced measures of red…

In the U.S., doctors who care for people with sickle cell disease (SCD) often don’t have a clear understanding of what motivates patients to participate in clinical trials, according to a survey-based study called LISTEN. Based on the findings, researchers offered a range of suggestions for how doctors can…

CSL is investigating two potential new treatments for sickle cell disease: An injected monoclonal antibody designed to prevent pain crises by reducing the neutrophil levels in the blood: Researchers are studying whether the monoclonal antibody treatment can decrease the number of pain crises and reduce the chances of long-term injury…

The U.S. Food and Drug Administration (FDA) has granted priority review — a decision that could speed a drug’s approval — to an application for the oral therapy mitapivat for sickle cell disease (SCD). According to developer Agios Pharmaceuticals, mitapivat, if approved, will be the first therapy of…

Casgevy (exagamglogene autotemcel) is now approved in the U.S. to treat children as young as 2 with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) initially approved the Vertex Pharmaceuticals’ gene-editing therapy in 2023 for SCD patients ages 12 and older who…