New evidence shows hematopoietic cell transplantation offers durable relief for sickle cell disease

New evidence shows hematopoietic cell transplantation offers durable relief for sickle cell disease

Patients who underwent hematopoietic cell transplantation for sickle cell disease saw high rates of survival without disease symptoms and low rates of severe side effects or complications years after their procedure, according to a new study. The study included over 1,000 patients, representing the largest and most comprehensive analysis of long-term transplant outcomes to date…

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Agios shares slump on sickle cell disease data

Agios shares slump on sickle cell disease data

Shares in Agios Pharma lost nearly half their value in pre-market trading today after it posted mixed clinical results from a phase 3 trial of its sickle cell disease (SCD) therapy mitapivat, sending its investors into a spin. In the pivotal RISE UP study, mitapivat – an orally-active pyruvate kinase (PKR) activator – was able…

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Gut microbial imbalance linked to chronic sickle cell disease pain

Gut microbial imbalance linked to chronic sickle cell disease pain

A research collaboration involving scientists and students at The University of Texas at Dallas has found a clear connection between chronic sickle cell disease pain and the bacteria present in the gastrointestinal tract. Dr. Katelyn Sadler, assistant professor of neuroscience in the School of Behavioral and Brain Sciences, is the corresponding author of the study, which was conducted in…

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