Search Results - "IFF, JOEL"

Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA by Iff, Joel, Zhong, Yi, Tuttle, Edward, Gupta, Deepshekhar, Paul, Xander, Erik Henricson

“…Aim: To evaluate treatment effects of eteplirsen among patients with Duchenne muscular dystrophy. Methods: Using real-world claims and electronic medical…”
Get full text

P260: Survival in eteplirsen-treated vs Duchenne muscular dystrophy natural history patients: An indirect treatment comparison using real-world data by O’Rourke, Erin, Iff, Joel, Done, Nicolae, Tuttle, Ed, Zhong, Yi, Wei, Fangzhou, Darras, Basil, McDonald, Craig, Mercuri, Eugenio, Muntoni, Francesco

Get full text

Survival among patients receiving eteplirsen for up to 8years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls by Iff, Joel, Done, Nicolae, Tuttle, Edward, Zhong, Yi, Fangzhou Wei, Darras, Basil T, McDonald, Craig M, Mercuri, Eugenio, Muntoni, Francesco

“…Introduction/AimsEteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip‐amenable variants, is associated with…”
Get full text

Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls by Iff, Joel, Done, Nicolae, Tuttle, Edward, Zhong, Yi, Wei, Fangzhou, Darras, Basil T., McDonald, Craig M., Mercuri, Eugenio, Muntoni, Francesco

“…Introduction/Aims Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip‐amenable variants, is associated with…”
Get full text

Delays in pulmonary decline in eteplirsen‐treated patients with Duchenne muscular dystrophy by Iff, Joel, Gerrits, Charles, Zhong, Yi, Tuttle, Edward, Birk, Erica, Zheng, Yeya, Paul, Xander, Henricson, Erik K., McDonald, Craig M.

“…Introduction/Aims Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States–approved treatment for…”
Get full text

Disease Progression Stages and Burden in Patients with Duchenne Muscular Dystrophy Using Administrative Claims Supplemented by Electronic Medical Records by Iff, Joel, Zhong, Yi, Gupta, Deepshekhar, Paul, Xander, Tuttle, Edward, Henricson, Erik, Schrader, Rachel

“…Introduction This study aims to identify stages of Duchenne muscular dystrophy (DMD) and assess the disease burden by progression stage using real-world…”
Get full text

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 by Craig, Joyce, Fuchs, Thomas, Jenks, Michelle, Fleetwood, Kelly, Franz, Dominik, Iff, Joel, Raschke, Michael

“…Purpose This analysis compared the rate of deep wound infections in patients with open tibia fractures, treated with intramedullary nails, receiving additional…”
Get full text

Delays in Progression of Duchenne Muscular Dystrophy with Eteplirsen Treatment: Attenuation of Pulmonary Function Decline and Projected Freedom from Continuous Ventilation (1398) by Iff, Joel, Gerrits, Charles, Birk, Erica, Tuttle, Ed, Zheng, Yeya, Henricson, Erik, McDonald, Craig

“…Abstract only…”
Get full text

Development of a Multivariable Proxy Model for Six-Minute Walk Distance (6MWD) in Duchenne Muscular Dystrophy (DMD) Using Machine Learning Methods (682) by Done, Nicolae, Iff, Joel, Signorovitch, James, Bertsimas, Dimitris, Henricson, Erik, Royer, Jimmy, McDonald, Craig

“…Abstract only…”
Get full text

Eteplirsen Treatment for Duchenne Muscular Dystrophy: A Qualitative Patient Experience Study by Iff, Joel, Carmichael, Chloe, McKee, Stephanie, Sehinovych, Ihor, McNeill, Carolyn, Tesi-Rocha, Carolina, Henricson, Erik, Muntoni, Francesco, Kitchen, Helen

“…Introduction Duchenne muscular dystrophy (DMD) is characterized by rapid functional decline. Current available treatment options aim to delay disease…”
Get full text

Differential Proteomic Analysis of STAT6 Knockout Mice Reveals New Regulatory Function in Liver Lipid Homeostasis by Iff, Joël, Wang, Wei, Sajic, Tatjana, Oudry, Nathalie, Gueneau, Estelle, Hopfgartner, Gérard, Varesio, Emmanuel, Szanto, Ildiko

“…Increased inflammatory signaling is a key feature of metabolic disorders. In this context, the role of increased pro-inflammatory signals has been extensively…”
Get full text

The Burden of Uncontrolled Glycemia—A Systematic Literature Review by FERNANDES, GAIL, IFF, JOEL, OVCINNIKOVA, OLGA, NASSIM, MARIA, SUN, LUCY, RICHARDS, MARGARET S., RAJPATHAK, SWAPNIL

“…The objective of this systematic literature review (SLR) was to understand the consequences of uncontrolled glycemia in patients with type 2 diabetes mellitus…”
Get full text

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy by Landfeldt, Erik, Iff, Joel, Henricson, Erik, Vishwanathan, V., Chidambaranathan, S., Biggar, W. Douglas, McAdam, Laura C., Mah, Jean K., Tulinius, Mar, Cnaan, Avital, Morgenroth, Lauren P., Leshner, Robert, TesiRocha, Carolina, Thangarajh, Mathula, Duong, Tina, Kornberg, Andrew, Ryan, Monique, Nevo, Yoram, Dubrovsky, Alberto, Clemens, Paula R., Abdel-Hamid, Hoda, Connolly, Anne M., Pestronk, Alan, Teasley, Jean, Bertorini, Tulio E., Webster, Richard, Kolski, Hanna, Kuntz, Nancy, Driscoll, Sherilyn, Bodensteiner, John B., Carlo, Jose, Gorni, Ksenija, Lotze, Timothy, Day, John W., Karachunski, Peter, Henricson, Erik K., Abresch, Richard T., Joyce, Nanette C., McDonald, Craig M.

“…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…”
Get full text