One-Shot “Miracle Cures” That Can Cost Millions

Gene therapies have been incredibly successful for many rare diseases, especially diseases of the blood and immune systems. For example, patients with severe combined immunodeficiency syndrome (SCID), commonly known as bubble boy disease, can be cured after receiving a bone marrow transplant using their own cells that have been genetically altered to add back the missing gene whose absence leads to the disease. However, patients, drug companies, and governments alike must grapple with the fact that these “miracle cures” are often exorbitantly priced.

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