AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial.

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AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial. | LitMetric

AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial.

Giovanni Baranello Francesco Muntoni

Gene Ther

The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, 30 Guilford Street, London, UK.

Published: November 2024

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Source

http://dx.doi.org/10.1038/s41434-024-00494-6 DOI Listing

Download full-text PDF	Source
http://dx.doi.org/10.1038/s41434-024-00494-6	DOI Listing

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