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SMA gene replacement therapy breakthrough

Nancy Kuntz, MD, Medical Director of Lurie Children’s Mazza Foundation Neuromuscular Program, was a principal investigator in a clinical trial of Zolgensma, a breakthrough, single-dose replacement therapy to treat children under age two with spinal muscular atrophy (SMA). The therapy was approved by the FDA in May 2019. SMA is a devastating genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Babies born with SMA often do not survive beyond two years of age.

Fetal EXIT procedure saves twins’ lives

Lurie Children’s fetal surgeon Aimen Shaaban, MD, Director of The Chicago Institute for Fetal Health, led a team of 40 multidisciplinary specialists in performing a rare EXIT procedure on twin girls. One twin had a large tumor on her neck that needed to be removed immediately. Additionally, she had a large build-up of fluid, which could compromise her sister’s health. Dr. Shaaban partially delivered the baby to gain access to remove the tumor while the healthier child remained in her mom’s womb. After the successful surgery, both babies were fully delivered.

Sculpting new ears

Akira Yamada, MD, PhD, an international expert in performing ear reconstruction surgery, particularly microtia or “small ear” reconstruction, joined the Division of Pediatric Plastic & Reconstructive Surgery in 2019. When reconstructing an ear, Dr. Yamada uses an innovative technique in which he creates a 3D framework of the ear using the patient’s own rib cartilage. His patients have shown tremendous improvement after this complex procedure.

First PCD Center of Excellence in Illinois

Lurie Children’s became one of only a handful of institutions in the U.S.—and the first in Illinois—to offer a Pediatric Primary Ciliary Dyskinesia (PCD) Center of Excellence. Directed by Terri Laguna, MD, MSCS, Division Head of Pulmonary and Sleep Medicine, the center serves as a regional referral and care location for children with a genetic disease that has a wide range of symptoms, ranging from lifelong respiratory disease to heart conditions and recurrent infections.

Progress reducing central line infections

A hospital-wide patient safety initiative to reduce the rate of central line associated blood stream infections (CLABSI) has made remarkable progress, decreasing the CLABSI standardized infection ratio from 0.9 at the start of the initiative in January 2018 to the current rate of 0.67 which represents a 26% decrease. The medical staff also achieved its goal of increasing the hospital’s CLABSI score as ranked by U.S.News & World Report from 3 to 4 out of 5. The initiative, using evidence-based practices, continues to be a major patient safety focus in FY20.