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ADDRESSING THE VALUE OF GENE THERAPY: ENHANCING PATIENT ACCESS TO TRANSFORMATIVE TREATMENTS

May 1, 2018

Gene therapy is a radical shift in our approach to disease treatment. By modifying the expression of a patient’s genes or repairing abnormal genes, gene therapy often addresses the root cause of diseases. While several new gene and cell therapies have received FDA approvals over the past 20 years, the field has recently experienced a turning point. Three gene therapies were approved for human medical use in the U.S. in 2017, including the first in the country for an inherited condition, and many more are likely on the way.

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