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Article

Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?

2 Apr 2014

Abstract excerpt

Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

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Identifiers

PMID: 24695683
DOI: 10.1126/scitranslmed.3008873

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Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?PMID 24695683Read ↗

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Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular DystrophySemantic proximity Read ↗

[Exon skipping therapy for Duchenne muscular dystrophy: outcome of preclinical study and prospective for clinical study].Semantic proximity Read ↗

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Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.Semantic proximity Read ↗

Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular AtrophySemantic proximity Read ↗

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