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Article

Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients.

Neuromuscular disorders : NMD - 1 Apr 2010

Helderman-van den Enden A T J M, Straathof C S M, Aartsma-Rus A, den Dunnen J T, Verbist B M, Bakker E, Verschuuren J J G M, Ginjaar H B

Abstract excerpt

Theoretically, 13% of patients with Duchenne muscular dystrophy may benefit from antisense-mediated skipping of exon 51 to restore the reading frame, which results in the production of a shortened dystrophin protein. We give a detailed description with longitudinal follow up of three patients wit...

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Source

Identifiers

PMID: 20153965
DOI: 10.1016/j.nmd.2010.01.013

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-20153965-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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