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…
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…
Lucy Beall's parents were expecting a fairly normal and straightforward birth. But when Lucy was born, the entire room was shocked. She weighed just six pounds and her skin…
Clinical trials are greatly beneficial in understanding more about therapeutics and various conditions. For example, the Phase 3 VIITAL trial will evaluate the safety, efficacy, and tolerability of EB-101, an…
People with epidermolysis bullosa (EB) often experience symptoms such as intense itching or severe pain. Unfortunately, these symptoms can be debilitating and may greatly affect overall quality-of-life. However, some burgeoning…
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…
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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…
The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…
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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