2024 - Clinic for Special Children

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Papers Published in 2024

Title: High-tech care in the Amish: Chronic respiratory failure in Ellis-van Creveld syndrome Published: June 18, 2024 Publication: Pediatric Pulmonology

Authors: Melissa O. Bowman, MD, Devyani Chowdhury, MD, Laura E. Poskitt, DO, Aaron Chidekel, MD

Title: Preemptive dual therapy for children at risk for infantile-onset spinal muscular atrophy Published: May 31, 2024 Publication: Annals of Clinical and Translational Neurology

Authors: Susan E. Matesanz, Karlla W. Brigatti, Millie Young, Sabrina W. Yum, Kevin A. Strauss

Abstract:

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.

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Our clinic serves as a trusted medical practice for children and adults facing rare genetic disorders. Our dedicated team works every day to prevent and treat genetic illnesses. Our facility is in the heart of the Amish and Mennonite communities in Lancaster County. Inside is filled with cutting-edge gene sequencing tools that allow us to deliver highly personalized care—a precise treatment option for the right patient at the right time.

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