Theragnosis for Duchenne Muscular Dystrophy.

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Publisher: Frontiers Research Foundation
Document Type: Article
Length: 403 words
Lexile Measure: 1310L

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Author(s): Leonela Luce, Micaela Carcione, Chiara Mazzanti, Paula I. Buonfiglio, Viviana Dalamà³n, Lilia Mesa, Alberto Dubrovsky, José Corderà­, Florencia Giliberto

Dystrophinopathies cover a spectrum of rare progressive X-linked muscle diseases, arising from <italic>DMD</italic> mutations. They are among the most common pediatric muscular dystrophies, being Duchenne muscular dystrophy (DMD) the most severe form. Despite the fact that there is still no cure for these serious diseases, unprecedented advances are being made for the development of therapies for DMD. Some of which are already conditionally approved: exon skipping and premature stop codon read-through. The present work aimed to characterize the mutational spectrum...

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Gale Document Number: GALE|A663969219