A cohort of African health ministers, with support from the African regional office of the World Health Organization (WHO), has opened a campaign to improve sickle cell disease (SCD) awareness, prevention, and care in that continent, where most SCD patients reside. The effort seeks to curb the blood disorder’s…
News
People with sickle cell anemia (SCA) — the most common and the most severe form of sickle cell disease (SCD) — are more commonly hospitalized in the cooler winter and spring seasons than in the hotter summer or fall, according to a study conducted in Brazil. Better understanding of…
Intravenous bisphosphonate therapy can ease bone pain in children with sickle cell disease (SCD), a new study reports. The study, “Sickle cell bone disease and response to intravenous bisphosphonates in children,” was published in the journal Osteoporosis International. Many people with SCD experience…
The first patient has been dosed in a Phase 1/2 trial assessing the safety and preliminary efficacy of Graphite Bio’s experimental gene editing therapy GPH101 (now called nula-cel) in people with sickle cell disease (SCD). The trial, called CEDAR (NCT04819841), is currently recruiting participants at three sites,…
Contraception practices and counseling for adolescent and young adult women with sickle cell disease (SCD) vary by the healthcare provider, especially among clinicians with more or less experience, a survey reported. Established clinicians were found to be more likely to provide contraceptive counseling than those still in training, by…
In a move to enhance three decades of treatment development in rare hematology, Pfizer will acquire Global Blood Therapeutics (GBT), a biopharmaceutical company that has been a leader in sickle cell disease (SCD) therapeutics, for $5.4 billion. The acquisition brings GBT’s treatment portfolio and pipeline — and its…
Children with sickle cell disease (SCD) exposed to higher doses of the oral therapy hydroxyurea report better blood-related clinical parameters than those with lower exposure, according to a new study from the U.S. The favorable outcomes were linked to higher adherence to treatment, suggesting that “adherence interventions have…
The gene editing therapy EDIT-301 appears to be working as intended in the first person with sickle cell disease (SCD) who received the experimental treatment, according to recent data from the Phase 1/2 RUBY clinical trial. Editas Medicine, which is developing EDIT-301, also announced that the U.S. Food…
Oxbryta (voxelotor) has been granted marketing approval in Great Britain to treat hemolytic anemia in people ages 12 and older with sickle cell disease (SCD). Eligible patients may take the once-daily oral therapy alone or in combination with hydroxyurea, a standard SCD therapy. The approval was granted…
The prevalence of a genetic disorder affecting red blood cells, called glucose-6-phosphate dehydrogenase (G6PD) deficiency, was equally high among people with sickle cell disease (SCD) and healthy blood donors in the Democratic Republic of Congo (DRC), a study has found. In addition, among SCD patients, the presence of G6PD…
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