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PGN-EDO51

FLevel 1 evidence

PGN-EDO51

Other/Research

Basic Information

Categories

Cell-penetrating peptide-PMO conjugates

Overview

PepGen investigational peptide-PMO conjugate for exon 51 skipping in Duchenne muscular dystrophy: Phase I results from year 2024 showed lower-than-expected exon-skipping efficiency, leading to discontinuation.

Efficacy Rating

Terminated, Duchenne Muscular Dystrophy, Exon Skipping
Evidence Level

F

Evidence Type

No data available

Confidence

No data available

China Regulatory Status

Cosmetic Ingredient Directory

No data available

Food/Supplements

No data available

Medicine

No data available

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Disclaimer

The content on this platform is for educational and informational purposes only and does not constitute medical advice, formulation guidance, or a guarantee of regulatory compliance. All efficacy claims are labeled with their evidence level; please consult a professional before making any decisions.