Medical management of muscle weakness in Duchenne muscular dystrophy.

ABSTRACT:

Introduction

Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens.

Methods

A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists' responses were collected using a modified Delphi approach.

Results

Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid.

Dis

SUBMITTER: Rivera SR 

PROVIDER: S-EPMC7571693 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature