Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study.

Introduction: Duchenne muscular dystrophy (DMD) is characterized by rapid functional decline. Current available treatment options aim to delay disease progression or stabilize physical function. To aid in healthcare providers' understanding of the symptoms of disease that impact patients' experience, this study explored children's physical functioning, activities of daily living (ADLs), and health-related quality of life (HRQoL) after receiving eteplirsen, a weekly infusion indicated for individuals with DMD with exon 51 skip-amenable mutations.

Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls.

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 overall survival in a US cohort receiving eteplirsen and contextualize these outcomes versus DMD NH.

Methods: US patients with DMD receiving eteplirsen were followed through a patient support program, with data collected on ages at eteplirsen initiation and death/end of follow-up.

Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA.

To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. Using real-world claims and electronic medical record data, this retrospective comparative analysis assessed eteplirsen-treated and control cohorts matched by age, disease progression state, and pre-index period healthcare resource utilization. Poisson regression was used to evaluate eteplirsen effects on healthcare resource utilization outcomes.

Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystrophy.

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 shown that eteplirsen is associated with a statistically significant attenuation of pulmonary decline.

Disease Progression Stages and Burden in Patients with Duchenne Muscular Dystrophy Using Administrative Claims Supplemented by Electronic Medical Records.

Introduction: This study aims to identify stages of Duchenne muscular dystrophy (DMD) and assess the disease burden by progression stage using real-world administrative claims supplemented by relevant electronic medical record (EMR) data.

Methods: Claims and EMR data from the Decision Resources Group's Real World Data Repository (2011-2020) were used to identify patients with DMD by diagnosis code and to stratify them into four disease stages by diagnosis and procedure markers reflective of DMD progression. Clinical and medical history data from the Cooperative International Neuromuscular Research Group (CINRG) were used to validate the developed claims-based staging algorithm.

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy.

Objectives: The objective of this study was to examine the psychometric properties of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) in Duchenne muscular dystrophy (DMD), a rare, severely debilitating, and ultimately fatal neuromuscular disease.

Systematic review and meta-analysis of the additional benefit of local prophylactic antibiotic therapy for infection rates in open tibia fractures treated with intramedullary nailing.

Purpose: This analysis compared the rate of deep wound infections in patients with open tibia fractures, treated with intramedullary nails, receiving additional locally-delivered antibiotics to those receiving standard care.

Methods: Two systematic literature searches identified studies reporting infection rates in patients treated with intramedullary nails for tibia fractures receiving systemic antibiotics only (search one) and in patients receiving adjunctive locally-administered antibiotics peri-operatively at the tissue-implant interface (search two). After applying inclusion and exclusion criteria, 14 and seven papers from searches one and two, respectively, were included in meta-analyses.

Differential proteomic analysis of STAT6 knockout mice reveals new regulatory function in liver lipid homeostasis.

Increased inflammatory signaling is a key feature of metabolic disorders. In this context, the role of increased pro-inflammatory signals has been extensively studied. By contrast, no efforts have been dedicated to study the contrasting scenario: the attenuation of anti-inflammatory signals and their role in metabolic homeostasis.