Similar disease progression in nonsense Duchenne muscular dystrophy boys as general natural history: Single Brazilian center 15 years registry view

Duchenne muscular dystrophy is a progressive and fatal X-linked neuromuscular disease. Emergent disease-modifying therapy (DMT) in nonsense Duchenne muscular dystrophy (nmDMD) has brought new perspectives to slow down functional decline in this fatal disease. To investigate if there are differences...

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Veröffentlicht in:	European journal of paediatric neurology 2024-11, Vol.53, p.117-122
Hauptverfasser:	Nardes, Flávia, Araújo, Alexandra Prufer de Queiroz Campos, Russi, Sofia, Henriques, Sarah Falcão Brasileiro
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Brazil - epidemiology Child Child, Preschool Codon, Nonsense Disease Progression Humans Infant Male Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - physiopathology Registries Retrospective Studies Young Adult
Online-Zugang:	Volltext
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