Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

Lindsay Alfano,Jay S. Charleston,Anne M. Connolly,Linda H. Cripe,C Donoghue,Robert Dracker,J. Dworzak,Helen Eliopoulos,D. Frank,Sarah Lewis,Karin Lucas,Jessie Lynch,Anthony J. Milici,Amy Flynt,Emily Naughton,Louise R. Rodino-Klapac,Zarife Sahenk,Frederick J. Schnell,G. David Young,Jerry R. Mendell,Linda Lowes

Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

2019

AbstractThis analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with DMD (N = 12) and confirmed genetic mutatio

Keywords:

Pediatrics
Eteplirsen
Physical therapy
Medicine
Diabetes mellitus
Duchenne muscular dystrophy
in patient
long term treatment

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