Bluebird bio claims first approval for gene therapy zynteglo estenosis lumbar ejercicios in eu – biopharma global – drug discovery news

The EU regulator has contractura lumbar izquierda given a conditional approval to Zynteglo, which is based on CD34+ stem cells taken from patients that are genetically modified to contain a working gene for beta-globin, a hemoglobin component that is deficient in thalassemia patients. Patients are given conditioning chemotherapy to clear their columna vertebral lumbar bone marrow of cells before the cells are reinfused.

The one-shot therapy – previously known as LentiGlobin – can now be used in the EU to treat patients aged 12 or over with transfusion-dependent thalassemia (TDT), in other words, those who are the most enfermedades lumbares severely affected by the disorder. Bluebird is carrying out a pair of phase 3 trials and a long-term follow-up study to try to confirm its safety and escoliosis lumbar efficacy.

That level of pricing would be in the same ballpark as allogeneic hematopoietic stem cell transplantation (HSCT), which can be attempted in TDT patients but unlike Zynteglo relies on finding a matched donor and also carries significant risks including treatment-related mortality, graft failure, graft versus host disease (GvHD) and opportunistic infections.

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