The impact of genotype on outcomes in individuals with Duchenne muscular dystrophy: A systematic review.

Szabo, Shelagh M; Gooch, Katherine L; Mickle, Alexis T; Salhany, Renna M; Connolly, Anne M

Szabo, Shelagh M; Gooch, Katherine L; Mickle, Alexis T; Salhany, Renna M; Connolly, Anne M.

Afiliação

Szabo SM; Broadstreet Heath Economics & Outcomes Research, Vancouver, British Columbia, Canada.
Gooch KL; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Mickle AT; Broadstreet Heath Economics & Outcomes Research, Vancouver, British Columbia, Canada.
Salhany RM; Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA.
Connolly AM; Division of Neurology, Nationwide Children's Hospital, Ohio State University, Columbus, Ohio, USA.

Muscle Nerve ; 65(3): 266-277, 2022 03.

Article em En | MEDLINE | ID: mdl-34878187

Assuntos

Distrofia Muscular de Duchenne; Criança; Distrofina/genética; Genótipo; Humanos; Volume Sistólico; Função Ventricular Esquerda

Palavras-chave

Duchenne muscular dystrophy; exon skipping; genotype; natural history; pathogenic variants

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Prognostic_studies / Systematic_reviews Limite: Child / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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