News

Health Canada has granted priority review to Chiesi Global Rare Diseases’ application seeking approval of Filsuvez (birch triterpenes), a topical gel to treat skin wounds in people with dystrophic epidermolysis bullosa (DEB) or junctional epidermolysis bullosa (JEB), ages 6 months or older. Priority review is granted to therapies for…

People with all subtypes of recessive dystrophic epidermolysis bullosa (RDEB) usually report that the skin-related disease has a substantial impact on their health-related quality of life, but a new study shows that the perceived impact of RDEB among patients tends to lessen over time. These findings suggest that,…

Parents reported that oral health problems lead to a poorer quality of life for their children with epidermolysis bullosa (EB), a study in Brazil shows. Overall, children with EB had more oral lesions, such as blisters and ulcers, and higher levels of gingival inflammation (inflammation of the gums) and…

Authorities in the U.K. have approved Krystal Biotech‘s gene therapy gel, Vyjuvek (beremagene geperpavec-svdt), to treat wounds in people with dystrophic epidermolysis bullosa (DEB) who have disease-causing mutations in the COL7A1 gene. The approval from the U.K.’s Medicines and Healthcare products…

From early school through adult life, people with epidermolysis bullosa (EB) consistently face peer discrimination, institutional inflexibility, employment challenges, and administrative burdens, according to patient interviews. Family support, understanding educators and colleagues, flexible work arrangements, and patient organizations emerged as the most meaningful facilitators of social participation in the…

Inmune Bio is expanding its partnership with the U.K.-based charity Anthony Nolan to advance an investigational stem cell therapy — dubbed CORDStrom — that’s primarily being developed to treat the rare skin-related disease RDEB, fully known as recessive dystrophic epidermolysis bullosa. Under the agreement, Anthony Nolan —…

In a new deal, Denmark-based Leo Pharma is acquiring Replay — and its “next-generation gene therapy platform” — with an eye toward developing a topical treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease causing extremely fragile skin. The gene therapy company, based in the U.S., is…

A study across seven European countries found that adults with epidermolysis bullosa (EB) report more pain and significantly worse health-related quality of life than the general population, with severe EB having the greatest day-to-day impact. “These results fill a critical evidence gap, supporting integration of EB-specific data into health…

A baby girl born with severe skin erosions and ulcers was diagnosed with a rare form of epidermolysis bullosa simplex (EBS) that carries the risk of heart disease, according to a case study that highlights the distinct skin features and potential heart risks of this EBS subtype. Caused by…

Wounds of people with epidermolysis bullosa (EB) are frequently colonized by antibiotic-resistant bacteria, which may contribute to poorer wound healing, according to a study from Australia. The scientists said their study, the first of its kind done in Australia, “provides valuable descriptive data on microbial composition and resistance patterns…

An 81-year-old man with junctional epidermolysis bullosa (JEB) has lived with recurrent blistering since childhood and developed multiple cancers over his lifetime, including skin cancers and urothelial carcinoma, a rare cancer of the urethra, a case study reports. Of the multiple skin cancers, most of them were squamous cell…

People with recessive dystrophic epidermolysis bullosa (RDEB) often experience a profound loss of nerve cells in the front of the eye, accompanied by increased numbers of inflammatory immune cells, a study found, suggesting that treatments to promote the health of these nerve cells and reduce eye inflammation may be…