Medical management of muscle weakness in Duchenne muscular dystrophy.
PLoS One
; 15(10): e0240687, 2020.
Article
em En
| MEDLINE
| ID: mdl-33075081
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. DISCUSSION: The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Corticosteroides
/
Debilidade Muscular
/
Distrofia Muscular de Duchenne
/
Morfolinos
Tipo de estudo:
Guideline
/
Qualitative_research
Limite:
Child
/
Humans
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article