Vyjuvek (beremagene geperpavec) successfully healed widespread skin wounds on an infant boy with severe dystrophic epidermolysis bullosa (DEB), according to a case study. Before treatment with gene therapy, the boy failed to thrive and achieve major motor milestones. After four months of weekly Vyjuvek, however, he developed motor…
A buildup of pro-inflammatory signaling molecules in skin wounds may explain the slow healing process experienced by people with recessive dystrophic epidermolysis bullosa (RDEB), a study suggested. The researchers said the findings support the development of therapies targeting wound-associated pro-inflammatory signals to prevent…
Treatment with dupilumab, a medicine approved for certain inflammatory disorders, eases itch and disease severity in people with different subtypes of dystrophic epidermolysis bullosa (DEB), a small study suggests. Dupilumab-related benefits seen in a woman before she stopped treatment and got pregnant were mostly maintained throughout her pregnancy and…
The stem cell therapy CORDStrom, in the pipeline at Inmune Bio, was found in a clinical trial to reduce itch and pain in children — from infants to teenagers — with intermediate and severe recessive dystrophic epidermolysis bullosa (RDEB), new data show. The treatment candidate also reduced disease…
A topical ointment containing diacerein, an arthritis medication, promoted wound healing in a 5-year-old girl with hard-to-treat recessive dystrophic epidermolysis bullosa (DEB), a case study reports. “These data characterize diacerein as a potential candidate for improving wound healing in RDEB through its impact on inflammatory [cells],” researchers wrote. The…
Adults with recessive dystrophic epidermolysis bullosa (RDEB) have abnormal immune cell profiles in blood and skin samples, a study reveals. The patients also had high blood levels of pro-inflammatory fat-like lipids and immune signaling proteins. These findings “underscore the concept of RDEB as [a] genetic disorder with distressed immunometabolism/inflammation,”…
Becoming a self-taught provider of home-based skin care is one of the many challenges faced by parents of children with epidermolysis bullosa (EB) — most of whom reported often being the primary caregiver for their affected sons and daughters in an interview-based study from Norway. Other challenges identified in…
Treatment with losartan, a commonly used oral blood pressure medication, safely reduced the signs and symptoms of recessive dystrophic epidermolysis bullosa (RDEB) in children, according to the results of a Phase 1/2 clinical trial. The repurposed medication particularly benefited young patients with severe disease, and was well tolerated with…
Two brothers shared a mild but distinct form of junctional epidermolysis bullosa (JEB) that affected the teeth, nails, and groin tissue, a case study reported. While there were no signs of new blisters, the brothers had chronic lesions characterized by delayed wound healing. The case study, “…
The experimental therapy ELX-02 reversed the effect of nonsense mutations in skin cells derived from people with recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB), a study found. Nonsense mutations in genes result in shorter, non-functional proteins, which commonly occur in RDEB and JEB patients, and…
C-reactive protein (CRP), a standard marker for inflammation, is an indicator of the severity of recessive dystrophic epidermolysis bullosa (RDEB) but not of active skin infections, according to a recent study of inflammatory biomarkers. “Our findings identify C-reactive protein as an excellent biomarker for disease severity in RDEB…. [and]…
The first patient has been dosed in a clinical trial evaluating Aegle Therapeutics‘ AGLE-102, an investigational topical treatment for dystrophic epidermolysis bullosa (DEB). The Phase 1/2a study (NCT04173650) is assessing the safety and efficacy of the therapy, applied topically to skin lesions, against a placebo in up…
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