News

The sickle disease of 83 percent of adolescents improved after 16 weeks of taking Global Blood Therapeutics‘ voxelotor, preliminary results of a Phase 2a clinical trial indicate. Another important finding was that voxelotor prevented irregularly shaped oxygen-carrying hemoglobin cells from clumping in more than half of those treated. The clumping prevents…

Prolong Pharmaceuticals has completed enrolling patients in an ongoing Phase 2 clinical trial of  Sanguinate, a treatment for the painful blocked blood vessel episodes that occur in sickle cell disease. The cause of the episodes, known as vaso-occlusive crises, is abnormally shaped red blood cells  bunching up and blocking…

Researchers have found that a new dosing regimen of the drug hydroxyurea results in levels of fetal hemoglobin above 20 percent in sickle cell anemia patients, and reduces hospitalizations by at least two-fold. Hydroxyurea has been shown to be of clinical benefit to children with sickle cell anemia (SCA) and is…

Vertex Pharmaceuticals and CRISPR Therapeutics recently announced a joint collaboration to co-develop and co-commercialize CTX001 as a gene-editing treatment of diseases of hemoglobin, including β-thalassemia and sickle cell disease (SCD). The announcement follows the presentation of preclinical data for CTX001 at the American Society of Hematology…

Intellia Therapeutics has used gene editing to replace an oxygen-carrying component in blood that is defective in sickle cell disease. The work involved editing the BCL11A gene in a mouse model of the disease to produce an effective version of the oxygen-carrying component, hemoglobin. Intellia discussed the project at the…

Sickle cell disease patients who have relatively low pain levels use opioids more when they are sad, anxious, or experience other negative emotions, a study indicates. The Johns Hopkins University research was based on entries in 45 patients’ electronic diaries. It suggested that patients misused opioids when they increased their intake…

A new online platform, called oneSCDvoice.com, will help those affected by sickle cell disease (SCD) by offering free access to comprehensive support and a robust knowledge network . rareLife solutions, the health tech, design and research team behind oneSCDvoice and other custom rare disease platforms, will launch the modular community…

Anyah Randolph, a teenager with sickle cell disease (SCD), was recently able to join her school class on a trip to Europe – and even climb the Eiffel Tower in Paris after the elevator broke – thanks to the help of hydroxyurea and Loyola Medicine pediatricians. Anyah had…

Following a review of studies published in the last decade, experts have updated their recommendations for managing a sickle cell disease-related eye complication known as sickle cell retinopathy (SCR). Their paper, “Sickle cell retinopathy: improving care with a multidisciplinary approach,” appeared in the Journal of Multidisciplinary Healthcare. SCR, a…

An independent review board has found Global Blood Therapeutics’ sickle cell disease treatment voxelotor safe, and recommended that its Phase 3 clinical trial continue. The hallmark of sickle cell disease is damage to a red blood cell protein called hemoglobin that carries oxygen to organs and tissue. The damage causes the cells…