News

A $100,000 grant from Global Blood Therapeutics (GBT) will help expand a novel nurse training program at the University of Tennessee Health Science Center (UTHSC) that aims to improve care for sickle cell disease (SCD) patients. The free training program — a “boot camp” for registered nurses —…

Reduced dietary iron eased blood vessel blockages and lessened organ damage in a mouse model of sickle cell disease (SCD), a new U.S.-based study reported. Low iron also reduced gut inflammation in SCD mice and restored a healthy balance of gut microbes. “Our study demonstrates for the…

People with sickle cell anemia are at a significantly higher risk of developing periodontitis, a serious gum disease, a study reported. In turn, periodontitis was associated with elevated levels of inflammation markers in patients and a three times greater chance of more than three painful vaso-occlusive crises (VOCs) each year.

The Qatar Ministry of Public Health has approved Endari (L-glutamine) to treat adults and children, starting at age 5, with sickle cell disease (SCD), the therapy’s developer, Emmaus Life Sciences, announced. “We are grateful that Qatari officials made access to Endari easier for the sickle cell patients…

The Sickle Cell Disease Association of America (SCDAA) has granted its President’s Award to board-certified nurse practitioner Genice Nelson, PhD,  of UConn Health’s New England Sickle Cell Institute (NESCI) Nelson was honored for her hard work and “immeasurable” dedication to the sickle cell community, and specifically for her…

A first patient has been enrolled in BEACON, a Phase 1/2 clinical trial into the safety and efficacy of BEAM-101, Beam Therapeutics’ transplant-based gene-editing cell therapy for sickle cell disease (SCD). Recruitment of adults with severe SCD, ages 18–35, continues at Boston Children’s Hospital in Massachusetts and…

A Phase 1b clinical trial testing the safety and pharmacological properties of the investigational oral medication FTX-6058 in people with sickle cell disease (SCD) is expected to continue enrolling patients through 2023, according to Fulcrum Therapeutics, the therapy’s developer. “We believe we are well positioned, with a…

Recruitment is now underway in BEACON, a Phase 1/2 U.S. trial testing BEAM-101, a transplant-based gene-editing cell therapy for sickle cell disease (SCD). The interventional study, in which all participants will be given a single dose of the experimental treatment, is expected to enroll up to 15 adults with…

Researchers have developed a method to detect and measure large unintended DNA modifications created by CRISPR-Cas9 gene-editing methods currently being investigated as treatments for sickle cell disease (SCD). High levels of unintended gene modifications that occurred at selected target sites demonstrate the need for a more careful evaluation of…

The risk of death from end-stage kidney disease (ESKD) has generally declined in the U.S. over the last 20 years — but not as much for people with sickle cell disease (SCD). Patients with the genetic disorder still have a higher risk of mortality than those without SCD,…