Grace Paul

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

S. Al-Zaidy, A. Simon Pickard, Kavitha Kotha et al. · Pediatric Pulmonology · 2018 · 219 citations · Full text

Medicine, Genetics, Pediatrics +13

Geographic variations in cystic fibrosis: An analysis of the U.S. CF Foundation Registry

Benjamin T. Kopp, Lisa Nicholson, Grace Paul et al. · Pediatric Pulmonology · 2015 · 31 citations

Social Sciences, Health Sciences, Clinical Epidemiology +14

Hypervitaminosis A with fulminant secondary intracranial hypertension following personalized medicine-based Elexacaftor/Tezacaftor/Ivacaftor initiation in a preadolescent with cystic fibrosis

B Wisniewski, Shawn C. Aylward, Catherine O. Jordan et al. · Journal of Cystic Fibrosis · 2022 · 30 citations

Medicine, Neurology, Pharmacotherapy +9

Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio

Megan A. Waldrop, Shannon Chagat, Michael A. Storey et al. · Neuromuscular Disorders · 2023 · 23 citations

Pharmacy, Pharmacotherapy, Immunotherapy +3

Elexacaftor-Tezacaftor-Ivacaftor Therapy for Chronic Sinus Disease in Cystic Fibrosis

Shahid Sheikh, Mai‐Lan Ho, Mariah Eisner et al. · JAMA Otolaryngology–Head & Neck Surgery · 2023 · 19 citations · Full text

Medicine, Clinical Sciences, Otolaryngology +17