Wiskott-Aldrich Syndrome Pediatric Research and Clinical Trials

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

Généthon, the not-for-profit biotherapy laboratory funded by the French Muscular Dystrophy Association (AFM), and Boston Children’s have initiated a partnership to conduct an exciting new gene therapy (also called gene transfer) clinical trial for Wiskott-Aldrich syndrome (WAS).

The U.S. portion of the trial is funded by the National Heart, Lung and Blood Institute (NHLBI) through its Gene Therapy Resource Program (GTRP). Principal investigators at Boston Children's are Sung-Yun Pai, MD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and Luigi Notarangelo, MD, director of the Research and Molecular Diagnosis Program on Primary Immunodeficiencies in the Division of Allergy and ImmunologyDavid A. Williams, MD, chief of the Division of Hematology/Oncology and director of Translational Research at Boston Children's, serves as sponsor-investigator.

For more information about the study, and to find out if your child may be eligible to participate, please contact David Williams, MD (email), Luigi Notarangelo, MD (email) or Sung-Yun Pai, MD (email).

Boston Children’s is so much more than a hospital—it’s a community of researchers, clinicians, administrators, support staff, innovators, teachers, patients and families, all working together to make the impossible possible. ”
- Sandra L. Fenwick, President and CEO

Boston Children's Hospital
300 Longwood Avenue, Boston, MA 02115
For Patients: 617-355-6000
For Referring Providers: 844-BCH-PEDS | 844-224-7337

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