A bone marrow transplant may help reverse heart damage in people with sickle cell disease (SCD), according to a study. The findings were published in a letter to the editor titled, “Impact of Hematopoietic Cell Transplantation on Myocardial Fibrosis in Young Patients with Sickle Cell Disease,” in…
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A novel small molecule called SR-18292, which acts to boost the production of fetal hemoglobin, was shown to reduce disease severity in a mouse model of sickle cell disease (SCD) and demonstrated benefits in other lab testing in a new study. “We’ve uncovered a promising approach that can offer…
A Massachusetts scientist has been awarded the seventh annual Trailblazer Prize for Clinician-Scientists by the Foundation for the National Institutes of Health (FNIH) for his research into understanding human blood cell development and how it’s altered in conditions such as sickle cell disease (SCD) — work that helped lead…
The U.S. Food and Drug Administration (FDA) gave the greenlight for Iomab-ACT, a potentially less toxic conditioning regimen, to be tested in people with sickle cell disease (SCD) undergoing a stem cell transplant. The study will be conducted in collaboration between Actinium Pharmaceuticals, which is developing Iomab-ACT,…
Hydroxyurea does not reduce ovarian reserve, or the quantity and quality of eggs in the ovaries, in girls and young women with sickle cell disease (SCD), according to a direct analysis of ovarian tissue. These findings suggest measures to preserve fertility may be unnecessary before hydroxyurea treatment. “This…
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…
Women with sickle cell disease who undergo a stem cell transplant can become pregnant and give birth to healthy children, a new study shows. “The results of this study show that female [stem cell transplant] recipients can achieve successful and safe pregnancies,” Katja Sockel, MD, lead study author at University…
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…
Seeking treatment for acute bouts of pain in hospitals and urgent care clinics is a significant source of distress for people with sickle cell disease (SCD), a report suggests. In interviews with SCD patients, scientists found patients’ distress went beyond the pain itself and included feelings of stigma and…
Supplementing with vitamin D for six months safely improved bone health and functional capacity in children and adolescents with sickle cell disease (SCD), a study finds. More studies are required to assess the “optimal dose, duration of supplementation, long-term [adverse events], and efficacy in different types of SCD, wrote…
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