Conference goals are to (1) gather families affected by WAS together in a nonmedical setting to share experiences with each other and present concerns to experts in immunology, BMT, and genetics; (2) review research achievements in WAS diagnosis, treatment (including BMT approaches and gene therapy), post-transplant outcomes (reconstitution and side effects of chemotherapy), and management of medical complications and family psychological issues; (3) develop and prioritize outstanding questions for future research; and (4) coordinate with planned efforts for a national WAS registry.
