Joel Iff
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View article: Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy
Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy Open
Background Duchenne muscular dystrophy (DMD) leads to dilated cardiomyopathy and heart failure during teenage years or young adulthood. Eteplirsen promotes dystrophin production through skipping of exon 51 of the DMD gene. Objective This a…
View article: Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study
Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study Open
This exploratory study indicated that most caregivers perceived improvements or maintenances in aspects of their child's physical functioning, ADLs, and HRQoL since eteplirsen initiation, which they perceived to be a positive outcome.
View article: Survival among patients receiving eteplirsen for up to 8 years for the treatment of <scp>Duchenne</scp> muscular dystrophy and contextualization with natural history controls
Survival among patients receiving eteplirsen for up to 8 years for the treatment of <span>Duchenne</span> muscular dystrophy and contextualization with natural history controls Open
Introduction/Aims Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip‐amenable variants, is associated with attenuated ambulatory/pulmonary decline versus DMD natural history (NH). We report…
View article: Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA
Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA Open
Aim: To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. Methods: Using real-world claims and electronic medical record data, this retrospective comparative analysis assessed eteplirsen-treated and …
View article: P260: Survival in eteplirsen-treated vs Duchenne muscular dystrophy natural history patients: An indirect treatment comparison using real-world data
P260: Survival in eteplirsen-treated vs Duchenne muscular dystrophy natural history patients: An indirect treatment comparison using real-world data Open
View article: Delays in pulmonary decline in eteplirsen‐treated patients with <scp>Duchenne</scp> muscular dystrophy
Delays in pulmonary decline in eteplirsen‐treated patients with <span>Duchenne</span> muscular dystrophy Open
Introduction/Aims Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States–approved treatment for patients with DMD and exon 51 skip‐amenable mutations. Previous analyses have sho…
View article: Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy
Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy Open