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Story perspectives

Collagen Disorder Trials Lacking, New Treatments Show Promise

11/6/2025

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Summary

Story summary

There is a notable lack of clinical trials for collagen disorders due to risk aversion and funding disparities.
Most current trials focus on dystrophic epidermolysis bullosa (DEB).
Emerging approaches such as Programmable Addition via Site-specific Targeting Elements (PASTE) and Oligonucleotide-Topoisomerase Inhibitors (OTIs) offer safer editing.
Vyjuvek has been approved, signaling progress in treating these rare diseases.

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Story summary

There is a notable lack of clinical trials for collagen disorders due to risk aversion and funding disparities.
Most current trials focus on dystrophic epidermolysis bullosa (DEB).
Emerging approaches such as Programmable Addition via Site-specific Targeting Elements (PASTE) and Oligonucleotide-Topoisomerase Inhibitors (OTIs) offer safer editing.
Vyjuvek has been approved, signaling progress in treating these rare diseases.