Severe Combined Immunodeficiency (SCID) Pediatric Research and Clinical Trials

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Contact the Division of Allergy and Immunology

At Boston Children's Hospital, our care is informed by our research, and our discoveries in the laboratory strengthen the care we provide at each child's bedside. Boston Children’s scientific research program is one of the largest and most active of any pediatric hospital in the world.

Boston Children’s has an ongoing gene therapy trial for the X-linked form of SCID (known as SCID-X1). The worldwide, multicenter study is sponsored in the United States by David Williams, MD, chief of Hematology/Oncology and director of Translational Research at Boston Children's.

The study is currently open for enrollment. To find out if your child may be eligible to participate, please contact the Dana-Farber/Boston Children's gene therapy program.

Touching lives from Argentina to Boston

Can you imagine how you’d feel if you couldn’t hold your child? Agustin Caceres’s parents could not come in physical contact with him as a baby because he had X-linked severe combined immunodeficiency (SCID-X1), better known as “bubble boy disease.” Read how Boston Children’s and the Dana Farber’s Cell Manipulation Core Facility were able to use gene therapy so he could be in touch with his family and the world again, literally and figuratively. Read more about how Agustin was treated through an international gene therapy trial.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

Boston Children's Hospital
300 Longwood Avenue, Boston, MA 02115
For Patients: 617-355-6000
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