Oxbryta (voxelotor), a daily oral therapy for hemolytic anemia associated with sickle cell disease (SCD), is now available to eligible patients in the U.K. under an early access program. It received a positive scientific opinion by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) under the Early…
Most children with sickle cell anemia (SCA), the most common and often the most severe form of sickle cell disease (SCD), do not receive preventive antibiotic treatment or participate in yearly stroke screenings that are recommended for those with the condition, according to a study based on U.S. data.
Fewer adults and children with sickle cell disease (SCD) have sought medical care for disease-related events during the COVID-19 pandemic, resulting in lower hospitalization rates, but longer hospital stays, according to data collected from a specialty center in Southern California that cares for a large number of SCD patients.
Even while hospitalized with one of the acute pain crises that sickle cell disease (SCD) patients frequently experience, it was common for Rena Grant to continue plugging away at her job as director of legislation for a South Carolina House of Representatives committee. Her courage and dedication — and the…
Blood vessel narrowing due to external or internal stimuli, and the resulting reduced blood flow, may be the main drivers of the painful vaso-occlusive crises experienced by people with sickle cell disease (SCD). Inducing stimuli for such blood vessel narrowing include mental stress, pain, and extreme temperatures, either low…
Sangamo Therapeutics is assuming all rights to SAR445136, an investigational gene-edited cell therapy co-developed with Sanofi and now in a Phase 1/2 trial in adults with severe sickle cell disease (SCD). Early trial data support the therapy’s safety and tolerability, and its effectiveness at raising levels of fetal hemoglobin in patients,…
A new formulation of Oxbryta (voxelotor) that is suitable for treating children as young as 4 is now available in the U.S. at specialty pharmacies. Late last year, the U.S. Food and Drug Administration (FDA) expanded the approval of Oxbryta to treat children with sickle cell disease (SCD)…
Researchers at the University of York in the U.K. have been awarded a $3.1 million (£2.3 million) grant to accelerate research supporting stem cell gene therapy to treat sickle cell disease (SCD). The grant was awarded by the nonprofit Bill & Melinda Gates Foundation to track genetic changes in…
Agios Pharmaceuticals has launched a Phase 2/3 trial called RISE UP to test its investigational therapy mitapivat (AG-348) in people with sickle cell disease (SCD). The trial (NCT05031780), which is not yet recruiting, aims to enroll about 237 people with SCD, ages 16 and older. To be…
The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…
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