Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies 90% (9 of 10) of patients were able to ...

Motixafortide alone and in combination with natalizumab (VLA-4 inhibitor) led to robust hematopoietic stem cell (HSC) mobilization predicting 16.9 million and 19.6 million cells/kg collected in a ...

For the roughly 30,000 Americans living with hemophilia and thousands more with rare enzyme deficiencies, treatment often ...