Fellowship · Columbus, OH
Sponsored by Nationwide Children's Hospital · Director Stephen Kirkby
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
JAMA neurology · 2021 · 231 citations · Richard Shell
Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double-blind, placebo-controlled phase 1/2 clinical study.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023 · 116 citations · Karen McCoy
Impact of elexacaftor-tezacaftor-ivacaftor on bacterial colonization and inflammatory responses in cystic fibrosis.
Pediatric pulmonology · 2023 · 76 citations · Shahid Sheikh, Courtney Gelpi, Karen McCoy
Nutritional considerations for a new era: A CF foundation position paper.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023 · 61 citations · Alpa Patel
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Muscle & nerve · 2024 · 69 citations · Richard Shell
Real-world disparities and ethical considerations with access to CFTR modulator drugs: Mind the gap!
Frontiers in pharmacology · 2023 · 61 citations · Grace R. Paul
Cystic Fibrosis Foundation Evidence-Based Guideline for the Management of CRMS/CFSPID.
Pediatrics · 2024 · 28 citations · Michael Smiley
Cystic fibrosis macrophage function and clinical outcomes after elexacaftor/tezacaftor/ivacaftor.
The European respiratory journal · 2023 · 51 citations · Benjamin Wisniewski, Karen McCoy