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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…

A research team is conducting two projects to learn more about neurological problems, including cognitive impairment and stroke, in people with sickle cell disease (SCD). Led by Hyacinth I. Hyacinth, PhD, at the University of Cincinnati, the team will focus on how various anti-inflammatory medications might act to prevent…

Note: This story has been updated July 19, 2022, to correct that voxelotor is approved in the U.S. to treat sickle cell in patients ages 4 and older. Global Blood Therapeutics, known as GBT, has initiated an expanded access program to make voxelotor, its daily oral therapy…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…