A lung ultrasound can aid in accurately diagnosing a serious respiratory complication called acute chest syndrome in children with sickle cell disease (SCD), a study reported. The study, “Point-of-care lung ultrasound is more reliable than chest X-ray for ruling out acute chest syndrome in sickle cell…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
A researcher will use a four-year, $1.63 million grant from the National Institutes of Health (NIH) to help set up a quality control system for CRISPR-based therapies aimed at certain genetic disorders, starting with sickle cell disease (SCD). The grant was awarded to Kiana Aran, PhD, an associate…
Adakveo (crizanlizumab), by Novartis, is now available on the U.K.’s National Health Service (NHS) for sickle cell disease (SCD) patients 16 and older. The new treatment, which is delivered by an into-the-vein infusion, can be used alone or as an add-on to hydroxyurea to help reduce the frequency of…
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,…
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