José Lopes, PhD, managing science editor —

Transplanting a patient’s own healthy skin to treat their skin ulcers was found to be safe and effective for three people with recessive dystrophic epidermolysis bullosa (RDEB) for more than one year, a clinical trial shows. The study, “Cultured epidermal autografts from clinically revertant skin as…

A compound called rigosertib effectively suppressed tumor growth and induced selective death of cancer cells both in patient-derived cells and in mice with squamous cell carcinoma (SCC) and mice with recessive dystrophic epidermolysis bullosa (RDEB), according to a study. Based on these preclinical results, a Phase 1/2 clinical…

A provisional diagnosis based on clinical information is more accurate than imaging of skin biopsies in newborns and infants with inherited epidermolysis bullosa (EB), according to a new study. The research, “A retrospective cohort study evaluating the accuracy of clinical diagnosis compared to immunofluorescence and electron…

Increased levels of a protein called decorin extended survival, reduced skin fibrosis (scarring) and delayed the development of paw deformities and other key recessive dystrophic epidermolysis bullosa (RDEB) manifestations in a mouse model. The study, “Decorin counteracts disease progression in mice with recessive dystrophic epidermolysis bullosa,”…

Diagnostic testing in patients with epidermolysis bullosa (EB) is more frequent in the most severe disease types, according to a large study in North America. The findings also revealed a delay between showing signs at birth and clinical diagnosis in a subset of patients with recessive dystrophic EB…

Abeona Therapeutics plans to start a Phase 3 trial in 2019 testing the cell therapy candidate EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB). The multi-center, randomized study, named VITAL, will compare treatment with EB-101 to untreated wounds in the same patient. In total, 10-15 patients…

A 1% diacerein ointment could be a safe and well-tolerated way to treat generalized severe epidermolysis bullosa simplex (EBS), according to a study involving two boys treated for four weeks. In vitro data suggested that the diacerein ointment was retained in the skin for a prolonged period of time. The study,…

Treatment with intravenous immunoglobulin (IVIG) and off-label rituximab can lead to complete remission of epidermolysis bullosa acquisita (EBA), according to a study that also illustrates the variable clinical and immunological presentations of EBA. The study, “Meta-analysis of the clinical and immunopathological characteristics and treatment outcomes in epidermolysis bullosa…

Treatment with a wound care ointment called Terrasil reduced pain, itching, and blistering in the wound area in a patient with a long history of junctional epidermolysis bullosa (JEB), according to a new case report. The study, “Epidermolysis bullosa: a case report,” published in the…

Treatment targeting a specific molecule involved in inflammation — called PI3K-delta — eased skin lesions and prevented blistering in mouse models of epidermolysis bullosa acquisita (EBA), according to a new study. The research, “Therapeutic Effect of a Novel Phosphatidylinositol-3-Kinase δ Inhibitor in Experimental Epidermolysis Bullosa Acquisita,”…

An enzyme called granzyme B (GzmB) may be a key mediator of blistering in patients with epidermolysis bullosa acquisita (EBA) and other autoimmune skin disorders, according to a Canadian study. These findings suggest that a single treatment strategy could be developed for different conditions. The research, “…

A Phase 1/2 clinical trial assessing investigational QR-313 as a treatment for dystrophic epidermolysis bullosa (DEB) has begun and is now enrolling participants, ProQR Therapeutics recently announced. The randomized, double-blind, placebo-controlled WINGS trial is recruiting up to eight participants with recessive DEB — a more severe form of the disease —…