SCD News Briefs

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SCD Independently Associated With Impaired Cognitive Function

Sickle cell disease (SCD) is known to be associated with a higher prevalence of sensorineural hearing loss (SNHL) and poor school performance compared to hemoglobin AA carriers, increasing the risk for cognitive impairment in these patients. Nonetheless, hearing impairment in the absence of SCD is also negatively associated with learning-related cognitive functions, leaving the association…

Routine Spirometry for Adults With SCD May Mitigate Decline

The annual decline in lung function is similar between sickle cell disease and cystic fibrosis, according to a new study published in Blood Advances. These findings support the importance of routine spirometry evaluation in adults with sickle cell disease. Even though there are no known therapies to stop the decline or increase lung function in adult…

Children With SCD May Face Increased Nutritional Challenges

Food insecurity and housing instability are 2 social determinants of health linked with poorer diet quality in children with sickle cell disease (SCD), according to a recent investigation published in Pediatric Blood & Cancer. The authors found that children with SCD who lived in environments with food insecurity and housing instability consumed more dairy and…

Deferiprone Provides a New Treatment Option for SCD

Researchers discovered that deferiprone and deferoxamine both have acceptable efficacy and safety profiles for treating transfusional iron overload in sickle cell disease (SCD) and other anemias, as published in Blood Advances. Many patients with SCD and other anemias are treated with chronic blood transfusions. The risk of this form of treatment is iron overload, which…

TALEN-Based Process Leads to Gene Correction in SCD Cells

TALEN-based gene editing was able to correct mutations in the HBB gene of hematopoietic stem and progenitor cells (HSPCs) taken from patients with sickle cell disease (SCD), as presented at the 63rd American Society for Hematology Annual Meeting & Exposition. The researchers found that TALGlobin01, an autologous HSPC-based gene therapy, was able to achieve up…

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Phase 1 Trial Results Suggest Possible Functional Cure for Sickle Cell Disease

A phase 1 double-blind, randomized, placebo-controlled clinical trial of FTX-6058 yielded induction of HBG messenger RNA (mRNA) in healthy adult volunteers after 14 days of once-daily treatment, Fulcrum Therapeutics, Inc. announced. Researchers observed a 5.6-fold induction of HBG mRNA in the 20 mg dose cohort and a 6.2-fold HBG mRNA induction in the 30 mg…

Newly Developed Multiplex Network Could Improve Rare Disease Research

Researchers in Austria developed a multiplex network by mapping genes and their interactions on multiple levels, which can help better identify genetic defects and study their consequences.  To build the network, the team led by Jörg Menche, PhD, adjunct principal investigator at the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences…

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