Wiskott-Aldrich Syndrome

At Children’s Hospital Boston, our care is informed by our research, and ourdiscoveries in the laboratory strengthen the care we provide at each child's bedside. Children’s 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 Children’s have initiated a partnership to conduct an exciting new gene therapy clinical trial for Wiskott-Aldrich syndrome. The U.S. portion of the trial will be funded by the National Heart, Lung and Blood Institute (NHLBI) through its Gene Therapy Resource Program (GTRP).

Principal investigators at Children's are Sung-Yun Pai, MD, in the Division of Hematology/Oncology, and Luigi Notarangelo, MD, director of the Research and Molecular Diagnosis Program on Primary Immunodeficiencies in the Division of Allergy and Immunology. David A. Williams, MD, chief of the Division of Hematology/Oncology and director of Translational Research at Children’s, will serve as sponsor-investigator.

For more information about the study, and to find out if your child may be eligible to participate, please contact dawilliams@childrens.harvard.edu, Luigi.Notarangelo@childrens.harvard.edu or Sung-Yun.Pai@childrens.harvard.edu.

ABOUT CLINICAL TRIALS

Children’s coordinates hundreds of clinical trials at any given time. Clinical trials are studies that may involve:

• evaluating the effectiveness of a new drug therapy
• testing a new diagnostic procedure or device
• examining a new treatment method for a particular condition
• taking a closer look at the causes and progression of specific diseases 

Taking part in a clinical trial at Children’s is entirely voluntary. Our team will be sure to fully address any questions you may have, and you may remove your child from the medical study at any time. 

Search current and upcoming clinical trials at Children’s

Search the National Institutes of Health’s list of clinical trials taking place around the world