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CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

24 Apr 2024

Innovative malaria prodrug targets liver, enhances efficacy while reducing toxicity, preclinical studies show

Tafenoquine, a prodrug that could fulfill the criteria for a mass eradication campaign.

23 Apr 2024

Intraoperative anemia linked to higher female mortality after heart bypass surgery

Women are at higher risk of death when undergoing heart bypass surgery than men. Researchers at Weill Cornell Medicine have determined that this disparity is mediated, to a large extent, by intraoperative anemia-;the loss of red blood cells during surgery.

13 Apr 2024

Study reveals unexpected vulnerability of lung immune cells to SARS-CoV-2

The lung-cell type that's most susceptible to infection by SARS-CoV-2, the virus that causes COVID-19, is not the one previously assumed to be most vulnerable.

10 Apr 2024

New malaria vaccine strain shows promise in preclinical trials

In a report published on March 21, 2024 in EMBO Molecular Medicine (A replication competent Plasmodium falciparum parasite completely attenuated by dual gene deletion) investigators at Seattle Children's Research Institute and Sanaria Inc. describe the development of a whole malaria parasite vaccine strain that infects the liver, develops to the late liver stage, and then gets completely stuck and cannot burst out of the liver to cause symptomatic blood infection.

2 Apr 2024

Unlocking clotting mechanisms in caterpillar hemolymph for medical use

Blood is a remarkable material: it must remain fluid inside blood vessels, yet clot as quickly as possible outside them, to stop bleeding. The chemical cascade that makes this possible is well understood for vertebrate blood.

30 Mar 2024

New imaging technique maps oxygen movement in the brain

The human brain consumes vast amounts of energy, which is almost exclusively generated from a form of metabolism that requires oxygen.

28 Mar 2024

Innovative multimodal, non-invasive brain monitoring to enhance ECMO patient care

Extracorporeal membrane oxygenation (ECMO) can be a life-saving therapy for patients with acute heart or lung failure.

14 Mar 2024

Study finds lack of preventative care for children with sickle cell anemia

Children with sickle cell anemia are vulnerable to serious infections and stroke, but many do not receive the preventative care that could help them stay healthier for longer, a Children's Hospital Los Angeles study found.

6 Mar 2024

Genetic study reveals insights into ancestry-associated differences in type 2 diabetes

The development and progression of type 2 diabetes are affected by numerous biological processes, such as the body's response to insulin, the health of insulin-producing pancreatic beta cells, and the functioning of metabolic pathways.

6 Mar 2024

Iron dysregulation identified as potential trigger for long COVID

Problems with iron levels in the blood and the body's ability to regulate this important nutrient as a result of SARS-CoV-2 infection could be a key trigger for long COVID, new research has discovered.

4 Mar 2024

Higher COVID-19 death risk found in sickle cell disease and trait patients

UdeM's Stéphanie Forté and co-researchers in Canada and Brazil have shown how people with sickle cell disease or sickle cell trait have a higher chance of dying from COVID-19 than the general population.

29 Feb 2024

UMass Amherst study targets aplastic anemia with cell retraining approach

Researchers at the University of Massachusetts Amherst recently released a first-of-its-kind study that focuses on the rare autoimmune disorder aplastic anemia to understand how a subset of cells might be trained to correct the overzealous immune response that can lead to fatal autoimmune disorders.

27 Feb 2024

Novel treatment for polycythemia vera shows promise in clinical trial

A novel treatment for polycythemia vera, a potentially fatal blood cancer, demonstrated the ability to control overproduction of red blood cells, the hallmark of this malignancy and many of its debilitating symptoms in a multi-center clinical trial led by the Icahn School of Medicine at Mount Sinai.

21 Feb 2024

Pain screening could improve care for youth with sickle cell disease

A recent study from researchers at University Hospitals (UH) Connor Whole Health and UH Rainbow Babies & Children's Hospital describes a quality improvement project where pain screening procedures were embedded within an outpatient pediatric sickle cell disease (SCD) clinic.

13 Feb 2024