Article

Recent Trends in Antisense Therapies for Duchenne muscular dystrophy

2023-01-09

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting 1/3500 boys globally, characterized by progressive muscle breakdown and eventual death with an average lifespan in the mid-late twenties. While no cure yet exists for DMD, gene and antisense therapies have been heavily explored in recent years to better treat this disease. Four antisense therapies have received conditional FDA...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 8fcc5ace-d9fc-5c1e-aaf5-89c79c63520b
DOI: 10.20944/preprints202301.0139.v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Recent Trends in Antisense Therapies for Duchenne muscular dystrophyDOI 10.20944/preprints202301.0139.v1Read

Therapeutic approaches for Duchenne muscular dystrophy.Semantic proximity Read

Exon skipping: a first in class strategy for Duchenne muscular dystrophySemantic proximity Read

Brogidirsen and Exon 44 Skipping for Duchenne Muscular Dystrophy: Advances and Challenges in RNA-Based TherapySemantic proximity Read

Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseasesSemantic proximity Read

Antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy: progress and challenges.Semantic proximity Read

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophySemantic proximity Read

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular DiseasesSemantic proximity Read

Molecular treatments in Duchenne muscular dystrophy.Semantic proximity Read

Antisense oligonucleotides and their applications in rare neurological diseasesSemantic proximity Read

Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic.Semantic proximity Read

Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.Semantic proximity Read

Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot.Semantic proximity Read