Compare efficacy of gene therapy alone (monotherapy) or in combination with an SMN2 augmentation agent (dual therapy) for treatment of children at risk for spinal muscular atrophy type 1.
Title: High-tech care in the Amish: Chronic respiratory failure in Ellis-van Creveld syndrome
Published: June 18, 2024
Publication: Pediatric Pulmonology
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Authors: Melissa O. Bowman, MD, Devyani Chowdhury, MD, Laura E. Poskitt, DO, Aaron Chidekel, MD