The U.S. Food and Drug Administration has granted orphan drug status to Sanofi’s rilzabrutinib, an investigational therapy aimed at preventing painful vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). This designation intends to encourage companies to develop therapies for rare diseases, or those affecting fewer…
News
An algorithm that grew out of a collaboration between different specialties at the Medical University of South Carolina (MUSC) may help reduce unnecessary hospital admissions for children with sickle cell disease (SCD), according to a new study. “This algorithm safely allows our care of these patients to align with…
Adolescents and young adults with sickle cell disease (SCD) may face challenges in accessing physical therapy that could help them manage symptoms, a study reports. While young people with poorer quality-of-life metrics tended to receive more referrals to physical therapists, this did not translate to more physical therapy visits…
Meningitis — an inflammation of the protective membranes surrounding the brain and spinal cord — remains associated with a case-fatality rate of nearly 30% in children with sickle cell disease (SCD), according to the findings of a new two-decade study from France. This is despite vaccinations for SCD children…
Many adolescents with sickle cell disease (SCD) aren’t having recommended depression screenings nor regularly visiting their primary care providers (PCPs), even though most have a registered PCP, a study reports. “Despite a higher overall rate of depression in this population … most youth are not being screened for depression…
A program that provides integrated primary and specialist care for adults with sickle cell disease (SCD) significantly reduced emergency department visits, according to a study from Massachusetts General Hospital. “Our Comprehensive Sickle Cell Disease Center (CSCDC) is one of few comprehensive adult care models that integrates [primary care physicians]…
Sickle cell disease (SCD) and sickle cell trait (SCT) can increase the risk of eye diseases associated with diabetes mellitus, according to a recent study. Findings indicated patients who had diabetes along with SCT or SCD were more likely to have procedures to treat eye disease and poorer overall…
Top-line data from the Phase 3 part of RISE UP, a clinical trial testing mitapivat in people with sickle cell disease (SCD), are expected by the end of the year, according to developer Agios Pharmaceuticals. If those results are positive and mitapivat wins regulatory approval for SCD, it…
Hydroxyurea, an approved medication to reduce the frequency of pain crises associated with sickle cell disease (SCD), may ease sleep-disordered breathing in children, perhaps by reducing inflammation, according to a study conducted at St. Jude Children’s Research Hospital, in Memphis. “Hydroxyurea therapy was associated with improved breathing…
An ongoing, early-phase clinical trial of pociredir, an oral small molecule being developed by Fulcrum Therapeutics as a possible treatment for sickle cell disease (SCD), is still recruiting adult patients at locations in the U.S. and one in South Africa. This Phase 1b trial, called PIONEER (NCT05169580),…
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