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At Pfizer Rare Disease, we define being “here” for the rare disease community as a commitment to listen, to learn, and to help make a difference in the lives of the more than 350 million people suffering from a rare disease worldwide.

For 30 years, we have focused on being present and a part of the journey rare disease families go through. We understand that every advance counts when it comes to bringing new medicines to those in need.

Of the 7,000 known rare diseases, less than 5% have an approved treatment option. These limited options have spurred a sense of urgency—to find new, potentially transformative approaches today.

At Pfizer Rare Disease, we are actively working to pioneer a new era of care in the field of gene therapy. Gene therapy works to address the underlying causes of certain genetic diseases using potentially one-time treatments. This potentially transformative technology is aimed at correcting or fixing a gene that may be defective.

“Through our research, we hope to unlock the potential of gene therapy to treat certain rare diseases and bring hope to those who are desperate for more options,” said Paul Levesque, Group President, Pfizer Rare Disease. “Leveraging cutting edge approaches, gene therapy could be significant for the next generation of people who are living with genetic diseases, making a meaningful difference in their lives—today and in the future.”

We are committed to making a lasting impact for the rare disease community through life changing innovations, trusted partnerships, and relentless passion. There is only one way we succeed—together. To celebrate Rare Disease Day and learn more about our work in gene therapy, visit our Gene Therapy page.