Search Results - "Iff, Joel"
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Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA
Published in Journal of comparative effectiveness research (01-09-2023)“…Aim: To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. Methods: Using real-world claims and electronic medical…”
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Survival among patients receiving eteplirsen for up to 8years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls
Published in Muscle & nerve (01-07-2024)“…Introduction/AimsEteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip‐amenable variants, is associated with…”
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Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls
Published in Muscle & nerve (01-07-2024)“…Introduction/Aims Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip‐amenable variants, is associated with…”
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Delays in pulmonary decline in eteplirsen‐treated patients with Duchenne muscular dystrophy
Published in Muscle & nerve (01-09-2022)“…Introduction/Aims Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States–approved treatment for…”
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Disease Progression Stages and Burden in Patients with Duchenne Muscular Dystrophy Using Administrative Claims Supplemented by Electronic Medical Records
Published in Advances in therapy (01-06-2022)“…Introduction This study aims to identify stages of Duchenne muscular dystrophy (DMD) and assess the disease burden by progression stage using real-world…”
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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
Published in International orthopaedics (01-05-2014)“…Purpose This analysis compared the rate of deep wound infections in patients with open tibia fractures, treated with intramedullary nails, receiving additional…”
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Development of a Multivariable Proxy Model for Six-Minute Walk Distance (6MWD) in Duchenne Muscular Dystrophy (DMD) Using Machine Learning Methods (682)
Published in Neurology (14-04-2020)“…Abstract only…”
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Delays in Progression of Duchenne Muscular Dystrophy with Eteplirsen Treatment: Attenuation of Pulmonary Function Decline and Projected Freedom from Continuous Ventilation (1398)
Published in Neurology (14-04-2020)“…Abstract only…”
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Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study
Published in Advances in therapy (01-08-2024)“…Introduction Duchenne muscular dystrophy (DMD) is characterized by rapid functional decline. Current available treatment options aim to delay disease…”
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Differential Proteomic Analysis of STAT6 Knockout Mice Reveals New Regulatory Function in Liver Lipid Homeostasis
Published in Journal of proteome research (01-10-2009)“…Increased inflammatory signaling is a key feature of metabolic disorders. In this context, the role of increased pro-inflammatory signals has been extensively…”
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The Burden of Uncontrolled Glycemia—A Systematic Literature Review
Published in Diabetes (New York, N.Y.) (01-07-2018)“…The objective of this systematic literature review (SLR) was to understand the consequences of uncontrolled glycemia in patients with type 2 diabetes mellitus…”
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Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy
Published in Value in health (01-10-2021)“…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…”
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