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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…
Tevin Coleman brought the snow inside last winter. Coleman, a running back for the New York Jets, his wife Akilah Coleman, and twins celebrated Christmas in January, to accommodate the last few weeks of the NFL regular season. His daughter, Nazaneen, 4, has sickle cell disease, and cold weather…
Two mouse models commonly used to study sickle cell disease (SCD) are not suitable for testing certain gene editing treatment approaches, including those for fetal hemoglobin production, because of their specific genetic makeup, a study reported. “This work provides a reminder for scientists to carefully consider the genetics of…
A novel strategy called Engineered Stem Cell Antibody Paired Evasion (ESCAPE) could be used to more safely prepare people with sickle cell disease (SCD) for experimental gene-editing approaches for treating the inherited blood disorder, according to researchers. ESCAPE may help patients ready for a bone marrow transplant, a key…
A public policy alliance has launched to advocate for federal policies that address the “crisis of care” facing the approximately 100,000 sickle cell disease (SCD) patients in the U.S. The ad-hoc alliance, called the Sickle Cell Disease Partnership, comprises more than a dozen groups representing patient advocates, healthcare…
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