Gene therapy for hemophilia gets FDA approval


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Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding.

A new drug emicizumab does not have clotting factors but can help people with hemophilia A.

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Drugmaker CSL Behring announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments. 

If this becomes a reality the reduced costs are only part of the benefits, treatments for the patients all come with risks and inconveniences, so lots of patients have great expectations of gene therapy for their quality of life.

Spinal muscular atrophy, sickle cell anemia, hemophilia and some kinds of cancer, we are in a time where gene therapies are making diseases that have plagued humanity something that can be done with one treatment that lasts for years or even all life.

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