Survival in Eteplirsen-treated vs Duchenne Muscular Dystrophy Natural History Patients: An Indirect Treatment Comparison Using Real-world Data
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Presenting Author - Erin O’Rourke, MS, CGC; Co-Author - Joel Iff, PhD, PharmD; Co-Author - Nicolae Done, PhD; Co-Author - Ed Tuttle, MBA; Co-Author - Yi Zhong, PhD; Co-Author - Fangzhou Wei, MS; Co-Author - Basil T. Darras, MD; Co-Author - Craig McDonald, MD; Co-Author - Eugenio Mercuri, MD; Co-Author - Francesco Muntoni, MD;
Meta Tag
Databases
Musculoskeletal system
Therapy
X-Inactivation/X-Linked Disease
Co-Author Joel Iff, PhD, PharmD
Co-Author Nicolae Done, PhD
Co-Author Ed Tuttle, MBA
Co-Author Yi Zhong, PhD
Co-Author Fangzhou Wei, MS
Co-Author Basil T. Darras, MD
Co-Author Craig McDonald, MD
Co-Author Eugenio Mercuri, MD
Co-Author Francesco Muntoni, MD
Presenting Author Erin O’Rourke, MS, CGC
Keywords
Duchenne muscular dystrophy
eteplirsen
survival
patient-level data
real-world data
mortality rates
age at treatment initiation
prognostic factors
selection bias
ambulatory and pulmonary function

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