The use of eculizumab, an immunosuppressive medication approved for several autoimmune diseases, was found to be safe and effective for treating hyperhemolysis syndrome or HHS — a serious complication of blood transfusions — in two pediatric patients with sickle cell disease (SCD), a study showed. HHS, which can be…
Emmaus Life Sciences, which markets Endari (L-glutamine) for sickle cell disease (SCD), has announced that it is complying with postmarketing safety regulations of the U.S. Food and Drug Administration (FDA). The announcement followed a 2.5-day routine inspection of Emmaus under the FDA’s Postmarketing Adverse Drug Experience…
The probability of developing organ and tissue damage — including in the lungs, kidneys, or eyes — increases as teenagers with sickle cell disease (SCD) transition from adolescence into adulthood, according to a study by researchers in France. Particularly, the study demonstrated that disease morbidity associated with chronic organ…
The Bahrain Oncology Center now is among the first in the world to offer Casgevy (exagamglogene autotemcel), an approved gene-editing therapy, to people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), a related blood disorder. That milestone, announced in a release by the country’s regulatory authority,…
Bluebird Bio has announced it’s completed collecting cells from the first sickle cell disease (SCD) patient receiving the gene therapy Lyfgenia (lovotibeglogene autotemcel), following its recent approval in the U.S. The cells were collected at Children’s National Hospital in Washington D.C., which is part of the company’s…
Having male adolescents with sickle cell disease (SCD) undergo fertility testing by semen analysis may not be feasible if it involves travel to a reproductive diagnostic center, according to a recent study. Despite having access to reproductive health education and compensation to cover transportation costs, only 15% of adolescents…
Novartis is recalling its sickle cell disease (SCD) treatment Adakveo (crizanlizumab) in the U.K., after the nation’s drug regulatory agency revoked the conditional approval it had previously granted to the antibody-based therapy. The U.K. Medicines & Healthcare products Regulatory Agency (MHRA) made its decision based on Adakveo’s failure…
Heavier menstrual bleeding and pain during menstrual periods appear to be prevalent among adolescents and young women with sickle cell disease (SCD), affecting their quality of life, but few of these patients use hormonal therapies to help regulate their cycles, a single-site U.S. survey study reported. Knowledge of what…
Skin grafts can ease pain associated with leg ulcers and improve wound healing in people with sickle cell disease (SCD), according to a study in France. The procedure consists of the surgical collection of a piece of healthy skin on the body that is then transplanted to the injured…
A portrait of 4-year-old Kynnedi Sturges, who is living with sickle cell disease (SCD) in Camden, Arkansas, was unveiled last week in an international art exhibit by the nonprofit Beyond the Diagnosis. Displaying artworks of 40 children with rare diseases, the exhibit is being hosted by…
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