Dr. Matthew Giefer, director of gastrointestinal endoscopy at Seattle Children's Hospital, and colleagues analyzed 342 children ages 0 - 18 with acute recurrent pancreatitis (ARP) and chronic pancreatitis (CP) from INSPPIRE (International Study Group of Pediatric Pancreatitis: In search for a cure), the nation's first and
only multicenter, National Institutes of Health - funded pediatric pancreatitis registry, led by Dr. Aliye Uc of University of Iowa Stead Family Children's Hospital.
For very rare diseases such as WAS,
multicenter clinical trials are the
only effective way of proving the safety and efficacy of gene therapy and having it rapidly approuved and made available to all patients.