News

The U.S. Food and Drug Administration (FDA) has delayed by three months its review deadline for Krystal Biotech’s gene therapy gel Vyjuvek (beremagene geperpavec) for dystrophic epidermolysis bullosa (DEB). The decision on Krystal’s application for approval of Vyjuvek, previously expected by mid-February this year, has been…

Blocking the activity of an immune cell receptor protein called FcyRIV prevented the development of epidermolysis bullosa acquisita in mice, a new study reports. The findings suggest this protein as a potential therapeutic target for this form of epidermolysis bullosa. The study, “Therapeutic effects of…

Disease activity may remain stable over years in people with epidermolysis bullosa, but will increase in the absence of treatment, according to a new report from a team of researchers in Australia. The team used the Epidermolysis Bullosa Disease Activity and Scarring Index, known as EBDASI — a…

An adenine base editor (ABE), a kind of molecular tool, was used to fix a disease-causing mutation in lab-grown fibroblasts from a person with dystrophic epidermolysis bullosa (DEB). The fix restored production of type VII collagen (C7), the protein missing in the disease, a study found. If the findings…

People with epidermolysis bullosa (EB) have an increased risk of delayed puberty and low bone mineral density (BMD), according to a medical records analysis. Increased incidence of delayed puberty with associated low BMD was notably seen in those with the recessive dystrophic epidermolysis bullosa (DEB) subtype. Predictors of…

The Venture into Cures virtual event raised more than $1.3 million for EB Research Partnership (EBRP), which seeks to advance research to cure epidermolysis bullosa (EB) and other rare diseases. In total, the three editions of the event, hosted by EBRP co-founders Jill and Eddie Vedder, have raised more…

Getting gene editing based on CRISPR, a kind of molecular scissors, delivered directly into cells may help restore the production of type VII collagen, the protein that’s missing in patients with dystrophic epidermolysis bullosa (DEB), a mouse study found. If the findings hold in humans, they “would allow the…

On Nov. 20, Jill and Eddie Vedder will host the third annual Venture Into Cures, a virtual show aimed at raising awareness for epidermolysis bullosa (EB) and funds for EB Research Partnership (EBRP). The event will feature stories about people with EB and their families, musical performances, and several…

EB-101, an investigational gene-corrected cell therapy, safely and effectively heals large, chronic wounds and eases pain in people with recessive dystrophic epidermolysis bullosa (RDEB), according to top-line data from the Phase 3 VIITAL trial. “We are very pleased with the topline results from our pivotal VIITAL study, which…

Pulsed dye laser therapy — a treatment commonly used for skin conditions such as certain types of birthmarks and scars — resulted in fewer blisters and eased erythema (reddening of the skin) in a 19-year-old girl with dominant dystrophic epidermolysis bullosa (DEB), according to a recent case report. The…

From sharing images and information on social media, to holding fundraisers to spread the word about epidermolysis bullosa (EB), supporters are set to mark National Epidermolysis Bullosa Awareness Week Oct. 25–31. “National Epidermolysis Bullosa Awareness Week … is a time to increase awareness of EB, to promote the need for a…

Many patients with dystrophic epidermolysis bullosa (DEB) need the help of a caregiver at home, and this adds up to their direct costs, which are higher than the mean costs of all patients with epidermolysis bullosa, according to a study covering five countries in Europe.  On average,…