News

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to IMR-687, Imara’s drug candidate for the treatment of sickle cell disease (SCD). The investigational therapy has also been granted orphan drug status by the FDA. Rare pediatric disease designation is granted to drugs that show promise…

GlycoMimetics, in collaboration with Pfizer, is now enrolling participants for a Phase 3 clinical trial to test the effectiveness and safety of the drug candidate rivipansel (GMI-1070) for the treatment of vaso-occlusive crisis (VOC) in hospitalized patients with sickle cell disease (SCD). “We can report that the Phase…

Differences in comorbidities, pain, healthcare utilization and psychosocial outcomes were addressed in three groups of sickle cell disease (SCD) patients, distinguished by their respective ages, in the Pain in the Sickle Cell Epidemiology Study (PiSCES). Other studies are needed to determine if age-specific healthcare measures might improve these patients’ lives. The…

Prolong Pharmaceuticals’ investigational drug for treatment of sickle cell disease (SCD)-associated complications, Sanguinate, showed promising results in patients experiencing vaso-occlusive crisis (VOC). According to the research study, “SANGUINATE Returns RBCs To More Normal Morphology In Patients With VOC,” presented at the 2017 Annual Symposium of the Foundation…

New guidelines for therapeutic strategies and pain management for sickle cell disease (SCD) should be considered by clinicians and healthcare teams, according to the author of a presentation made at the American College of Physicians (ACP) Internal Medicine Annual Meeting in San Diego. Controlling all the symptoms of SCD…

Patients with sickle-cell anemia have more eye blood flow resistance than those without the disease — a symptom closely linked to hemolysis, or rupture of red blood cells. This can lead to a better understanding of the disease as well as improved classification and care for sickle-cell patients. The Brazilian study, “Doppler velocimetry of…

Brazilian researchers suggest that a new clinically relevant subgroup of sickle cell disease (SCD) should be considered, based on hematological and biochemical parameters in patients with SCD. Determination of classical biomarkers and lipid parameters can contribute to a better definition of this complex disease and improve patient’s clinical care, they concluded. Their…

Patients who suffered a stroke and have sickle cell disease (SCD) can safely receive lytic therapy with tissue plasminogen activator (tPA), according to new research. The study, “Coexistent Sickle Cell Disease Has No Impact on the Safety or Outcome of Lytic Therapy in Acute Ischemic Stroke,” was published in…

Researchers have provided more evidence of the accuracy of a recently developed gene-editing technique, showing that the method precisely induces site-specific DNA alterations in the genome. The study may have implications for gene therapy for conditions such as sickle cell anemia. The paper, “Genome-wide target specificities of CRISPR RNA-guided…

The inaugural World Cord Blood Day on Nov. 15 this year will educate parents and healthcare professionals about the current uses for cord blood transplants, a non-controversial source of stem cells which have the ability to treat more than 80 life-threatening diseases, including sickle cell anemia. To date, more…