A recent milestone in gene therapy is transforming the outlook for people with rare genetic skin disorders, signaling a new era of personalized medicine and hope for patients who previously…
For families affected by rare genetic skin diseases, hope once meant daily bandages and vigilant wound care. That began to change in 2023, when US regulators approved the first-ever gene…
Krystal Biotech has just announced results from their Phase 3 trial called GEM-3, and the results were all positive! This trial investigated bermemagene geperpavec (B-VEC), or VYJUVEKTM as a potential therapy…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The FDA has recently granted the Fast Track designation to AGLE-102, a treatment for dystrophic epidermolysis bullosa. This designation will allow for the treatment to reach patients at a quicker…
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Dystrophic epidermolysis bullosa (DEB) is a condition that has many sub-types, one of which is pretibial. The COL7A1 gene, which is responsible for producing VII collagen, is the cause of…
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