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Pfizer Rare Disease is working to target the underlying causes of certain genetic diseases by focusing on highly specialized, potentially one-time gene therapy treatments.

Genes play an essential role in determining the function of each cell in the body, making up 30 million codes of DNA. If even one of these codes is damaged, a genetic alteration may occur causing a genetic disease, some of which can be debilitating and life-threatening. The goal of gene therapy is to restore normal function in affected tissues or cells, potentially enabling a patient to manage his or her disease without the need for ongoing treatments.

While the potential of gene therapy is exciting, there are still many unknowns. While gene therapy holds promise for people with genetic diseases, it will not be an appropriate solution for every patient. Clinical trials are underway to explore potential of gene therapy, including how to identify patients; the efficacy, safety and duration of response, and the impact of immune responses on patients.

"At Pfizer, we believe that gene therapy research holds tremendous possibility in bringing improved quality of life for patients,” said Bob Smith, Senior Vice President of the Global Gene Therapy Business. “Through our investment in research and development, and our continued commitment to innovative collaborations, we are working to make that promise a reality."

To highlight the scientific and technological advances that have led to the development of gene therapies, Pfizer Rare Disease has created an educational video that offers a glimpse into this potentially transformative treatment option, and the future impact it may have on the lives of those living with one of the nearly 6,000 rare genetic diseases worldwide.

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