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…
News
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…
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…
TALEN, a gene-editing technology being used by the clinical-stage biopharmaceutical company Cellectis to develop potentially life-changing therapies, was able to correct the mutation that causes sickle cell disease (SCD) in patient cells. That’s according to a new study published by researchers from the company, along with colleagues from…
Recent Posts
- When a sickle cell pain crisis interrupts a stretch of good health, part 1
- Scientists discover how stressed red blood cells make hemoglobin
- How my sickle cell disease advocacy has evolved over time
- New study links sickle cell anemia to lower ovarian reserve in women
- $3M Breakthrough Prize honors duo whose work changed SCD treatment
- Etavopivat Phase 3 results support potential approval in sickle cell disease
- New diagnoses result in new grief and a new relationship with my body
- Study finds lower treatment use among SCD patients in sub-Saharan Africa
- Outside, looking in: The silent isolation of living with sickle cell disease
- Bias in sickle cell care may be tied to opioid stigma, new study finds