Healio Psoriatic Disease recently reported that Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy designed for people with dystrophic epidermolysis bullosa (DEB), was granted Orphan Drug designation (ODD) by the Japanese…
In May 2023, the U.S. Food and Drug Administration (FDA) approved Vyjuvek, a gene therapy, for dystrophic epidermolysis bullosa (DEB). This treatment has brought support to numerous people living with…
Managing dystrophic epidermolysis bullosa (DEB) can be difficult. DEB is a rare genetic condition which causes fragile, easily blistered skin. There are multiple DEB subtypes with their own inheritance…
Those with recessive dystrophic epidermolysis bullosa (RDEB) have fragile, easily blistered skin. Unfortunately, because of dysfunctional tissue repair, wounds may take longer to heal – causing increased levels of scarring…
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…
CONTENT WARNING: Severe skin wounds and damage Working in rare disease is always hard- It means working in suffering and grief, but even with all our experience, we had to…
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…
The first patient has been dosed in the third phase of the VIITAL study, which is intended to evaluate the effectiveness of EB-101. This medication is a gene-corrected cell therapy…
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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…
According to a story from pm360online.com, the drug development company ProQR Therapeutics N.V. will be partnering with EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF) in order to…
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