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What if I live far away from a clinical trial site?

We understand that participating in a clinical trial commonly involves travel and a time commitment. As we design our clinical trials, we discuss them with patients, caregivers, and advocates, in order to minimize the burden for participants and their families.

Pfizer may provide study-related logistics services such as transportation. Because each study is different, we encourage you to ask the study team any questions you have. You can also refer to the informed consent document for the details about your study.

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What is gene therapy?

Gene therapy targets the underlying cause of a genetic disease. In gene therapy, a functioning gene is delivered to a targeted tissue in the body, enabling the cell or tissue to produce a protein that is either missing or is not functioning. This type of treatment has the potential to be used instead of drugs or surgery.

The safety and efficacy of gene therapy is being studied in clinical trials.

We believe that gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of disease and of ongoing treatments for the people who are affected by rare diseases.

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