News

A U.S.-based biotech company announced that it has reached a “significant” milestone in advancing its experimental stem cell treatment for recessive dystrophic epidermolysis bullosa (RDEB), a rare condition marked by extremely fragile skin. Inmune Bio said it has readied its process for commercial-grade manufacturing of CORDStrom, the stem…

A new biotechnology company called Rinascera Therapeutics has launched to develop therapies for people with epidermolysis bullosa and other rare genetic skin diseases. The company is working to advance two treatment candidates. RIN-002 is being developed for dystrophic epidermolysis bullosa (DEB), while RIN-001 is being developed for another…

A young girl with genetically confirmed epidermolysis bullosa simplex experienced a marked reduction in skin blistering — with no new sores for several months — with use of isotretinoin, an oral medication derived from vitamin A, according to a new case report. Its researchers say the child’s case…

Biomendics’ BM-3103, formulated as TolaSure Gel for epidermolysis bullosa simplex (EBS), has been granted fast track status by the U.S. Food and Drug Administration (FDA) for treating people with fragile skin due to EBS. The new designation could accelerate the development and review of the topical treatment if the gel…

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…