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Duchenne expert physician perspectives on Duchenne newborn screening and early Duchenne care

Duchenne muscular dystrophy (DMD) is a progressive, fatal neuromuscular disorder typically diagnosed between 4 and 5 years of age. DMD currently has five FDA approved therapies, which has led to increased interest in newborn screening (NBS) for DMD. Our objective was to explore the perspectives and...

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Detalles Bibliográficos
Autores principales: Armstrong, Niki, Schrader, Rachel, Fischer, Ryan, Crossnohere, Norah
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9804401/
https://www.ncbi.nlm.nih.gov/pubmed/35932090
http://dx.doi.org/10.1002/ajmg.c.31993