Sickle Cell Disease Insights

Orna & Vertex Partner on Gene Editing Therapies for SCD Patients

Orna Therapeutics and Vertex Pharmaceuticals are collaborating to develop gene-editing therapies for sickle cell disease, aiming to correct the genetic defect that causes the condition. This partnership combines Orna's RNA technology with Vertex's gene-editing expertise to potentially offer a new treatment option for patients.

Sickle Cell Disease's Impact on Mental Health

Living with sickle cell disease can significantly impact a person's mental health, leading to anxiety, depression, and other psychological challenges due to the chronic pain and frequent hospitalizations. Managing mental health is crucial for overall well-being in individuals with sickle cell disease.

When to Use Blood Transfusions in Sickle Cell Patients: Single Center Study

Blood transfusions and exchange transfusions are crucial treatments for managing sickle cell disease, especially during acute crises or to prevent complications. This study shares a single center's experience with these procedures, highlighting their indications and outcomes.

High Altitude Exposure Leads to Splenic Infarction in Sickle Cell Trait Patient

A patient with sickle cell trait experienced splenic infarction after being exposed to high altitude, highlighting the risk of such complications in individuals with this condition. The case underscores the importance of monitoring and managing health during high-altitude travel for those with sickle cell trait.

Recent studies have shed light on various aspects of Sickle Cell Disease (SCD). Afranie‐Sakyi et al. (2025) found that adults with SCD are experiencing early mortality, with a median age of death at 44 years, despite advancements in care. Notably, less than half of the patients were prescribed hydroxyurea, a medication known to reduce SCD complications. This highlights the need for better access to and education about disease-modifying treatments.

In another study, Sinha et al. (2025) observed the spontaneous emergence of autoantibodies associated with systemic lupus erythematosus (SLE) in a patient with SCD, suggesting that SCD may increase the risk for broader autoimmunity.

Lastly, a study by Reddy et al. (2025) is investigating the efficacy and safety of ketorolac compared to morphine for managing severe vaso-occlusive crises in children with SCD, which could potentially offer alternative pain management strategies.