Forest Ray PhD,  —

An open-label, Phase 1/2 clinical trial will evaluate the safety and efficacy of rigosertib in treating recessive dystrophic epidermolysis bullosa (RDEB) in people with an aggressive skin cancer called cutaneous squamous cell carcinoma (SCC). RDEB is known to bring a higher risk of developing SCC, which can be life-threatening. The…

A new and easy-to-use algorithm may help physicians diagnose epidermolysis bullosa aquisita (EBA) sooner, a process that now can take months or even years. In a recent study, researchers used artificial intelligence to analyze entire skin biopsies, of which the human eye normally can observe only a small part.

Ways to improve the quality of life for people with epidermolysis bullosa (EB) are needed, as those living with the disorder report a range of psychological and social impacts. In a series of interviews, a group of children and young adults with EB described their disease as significantly affecting…

A distinct set of bacteria colonizes the skin in and around wounds in people with dystrophic epidermolysis bullosa (DEB), according to a new study. The finding suggests that these patients may benefit from therapies that specifically target those bacteria and that encourage the growth of non wound-associated bacteria instead.

The first patient has been dosed in a Phase 2 trial investigating rigosertib as a treatment for cases of recessive dystrophic epidermolysis bullosa (RDEB) involving an aggressive skin cancer called squamous cell carcinoma (SCC). RDEB patients are at high risk of developing SCC. “We are pleased with the…

Krystal Biotech announced the full enrollment of a pivotal Phase 3 clinical trial of its topical gene therapy B-VEC (beremagene geperpavec) as a skin wound treatment for people with dystrophic epidermolysis bullosa (DEB). Top-line results on the 31 patients in the randomized GEM-3 trial (NCT04491604), a mix of children…

Skin-derived stem cells producing the ABCB5 protein outperformed those derived from bone marrow at migrating toward and integrating into wounds associated with recessive dystrophic epidermolysis bullosa (RDEB), a study reports. These cells also show a less pro-inflammatory potential, and their ability to modify the immune response may make them…

Abeona Therapeutics will proceed with enrollment for its pivotal Phase 3 study of EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB), following a meeting with the U.S. Food and Drug Administration (FDA). “We appreciate the clarity provided by the FDA and we are pleased to be…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AP103, a potential topical gene therapy for dystrophic epidermolysis bullosa (DEB). “Receiving an orphan drug designation from the FDA for our gene-therapy candidate, AP103, is a significant development for patients suffering…

Tonight at 8 p.m. EST, Jill and Eddie Vedder will host Venture Into Cures, a virtual event to raise awareness of epidermolysis bullosa (EB), and also raise funds through the stories of individuals and families living with the disorder and a series of celebrity performances and special appearances.

Filsuvez, a topical gel by Amryt Pharma, raises the probability of complete wound closure while reducing the time that wounds take to heal in people with epidermolysis bullosa (EB), new data from a Phase 3 clinical trial show. Investigators found a statistically significant 41.3% of patients treated Filsuvez had…