Exagamglogene autotemcel (Casgevy) ist die derzeit einzige zugelassene Geneditierungstherapie zur Einmalbehandlung der ...

Sickle cell disease (SCD) is a genetic disorder caused by a single point mutation in the β-globin gene, leading to the production of abnormal hemoglobin. Patients with SCD lacking a compatible donor ...

PYRUKYND is currently approved in Europe for adults with PK deficiency; thalassemia would represent its second indication, ...

About PYRUKYND® (mitapivat) U.S. INDICATION PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

How come food enters your body in so many different and attractive colours, but always comes out brown? And why does water enter your body colourless, but always come out yellow? The cause is the same ...

The evolution of biomedical science can be appreciated through studies of hemoglobin, the oxygen-carrying protein in red cells. Before molecular cloning, the geneticist Arno Motulsky noted, “Many ...

Would you advise this injection for an almost 5yr old boy who constantly struggles with his health? We have done extensive tests and only found an iron deficiancy (he' s been on ferrimed, jungle vit c ...

What is the MCHC Blood Test? A mean corpuscular hemoglobin concentration (MCHC) blood test measures the amount of a protein called hemoglobin in your red blood cells. It’s one of many measurements ...

This is a preview. Log in through your library . Journal Information PNAS is the world's most-cited multidisciplinary scientific serial. It publishes high-impact research reports, commentaries, ...

The targeted protein degrader BMS-986470 from Bristol Myers Squibb (BMS) boosts levels of fetal hemoglobin in cells, mice, and monkeys, the company reported this weekend at the American Society of ...