News

An imbalance of microbes in the digestive tract was found to contribute to bone disease in a mouse model study of sickle cell disease. Researchers discovered that transplanting healthy gut microbes protected the animals against SCD-related bone loss by increasing the levels of a bone growth hormone, which rose in…

New preclinical data support the ability of the ESCAPE platform, an approach Beam Therapeutics is advancing, to more safely prepare people with sickle cell disease (SCD) for gene-editing treatments now in development. Notably, these studies represent the first in vivo data for ESCAPE — that is, the experimental platform’s first evaluation in living…

A lung ultrasound may help in diagnosing acute chest syndrome (ACS), a serious lung complication in sickle cell disease (SCD), a pooled analysis of studies suggests. In fact, according to researchers, a lung ultrasound may be a better option than repeated chest X-rays to support an ACS diagnosis. An…

The experimental gene-editing therapy EDIT-301 appears to be working largely as expected in the first two sickle cell disease (SCD) patients treated as part of the Phase 1/2 RUBY trial, according to new data shared by the therapy’s developer, Editas Medicine. “These promising clinical results from the RUBY…

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…