Young people with sickle cell disease (SCD) who live in areas with more air pollution are more likely to be hospitalized due to acute chest syndrome (ACS), an SCD complication marked by symptoms that can include chest pain and difficulty breathing, a study found. “As ACS is a…
Researchers have used an existing noninvasive optical tool that leverages the way light interacts with tissue to detect disease in the small blood vessels in the brain, according to a study. This neurological complication of sickle cell disease (SCD) is linked to cognitive impairment. Used in a variety of…
Oral infections carry high risks for people with sickle cell disease (SCD), but most young patients who have insurance through Medicaid don’t receive dental care, a study suggests. Rates of preventive dental care and dental treatment were comparable between young sickle cell patients and young people without the disease, …
An intervention designed to improve adherence to hydroxyurea — by providing extra support from community health workers (CHWs) and daily text reminders — may enhance quality of life for adolescents with sickle cell disease (SCD), though the benefits may fade without continued support. The findings came from…
People with severe sickle cell disease (SCD) have reported significant improvements in their quality of life after receiving the approved gene-editing therapy Casgevy (exagamglogene autotemcel). That’s according to a new analysis of data from patients involved in a clinical trial that had tested the SCD treatment in individuals…
Correctsequence Therapeutics’ experimental gene-editing therapy CS-101 has been used to treat a person with sickle cell disease (SCD) for the first time. Prior to treatment with CS-101, the patient — a 21-year-old woman from Nigeria — had been experiencing frequent vaso-occlusive crises (VOCs), or painful episodes caused by…
Fundraising walks, a golf tournament, and a virtual blood drive are among activities planned to raise awareness of sickle cell disease (SCD) in September, National Sickle Cell Awareness Month. The campaign aims to raise funding for research into the inherited blood disorder and to support the approximately 100,000 U.S. residents…
Oryzon Genomics said it received European Medicines Agency approval to start a Phase 1b clinical trial of iadademstat in people with sickle cell disease (SCD). The trial — RESTORE (2025-521838-29-00), short for REgulation of Sickling ThrOugh Reprogramming Epigenetics — will enroll 40 SCD patients, ages 18 and…
Pfizer’s investigational therapy inclacumab failed to outperform a placebo in reducing the rate of vaso-occlusive crises — episodes marked by painful inflammatory responses — in people with sickle cell disease (SCD), according to new data from the Phase 3 THRIVE-131 study. “We recognize this news is disappointing…
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Beam Therapeutic’s BEAM-101, a one-time, gene-edited cell therapy for severe sickle cell disease (SCD). RMAT status is granted to therapies that have shown potential for treating serious or life-threatening conditions. It provides a…
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