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

To test the hypothesis that the folding pathways of evolutionarily related proteins with similar three-dimensional structures but widely different sequences should be similar, the folding pathway of ...

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 ...

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 ...

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