Steve Bryson, PhD,  science writer—

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…

An altered modification of proteins involved in controlling gene activity, called histones, may contribute to how severe the buildup of excessive scar tissue is in recessive dystrophic epidermolysis bullosa (RDEB), a study suggests. Blocking abnormal histone modification with the approved medicine valproic acid reduced the signs of scarring, or…

Daclatasvir, an approved antiviral medicine, safely reduced the signs and symptoms of recessive dystrophic epidermolysis bullosa (RDEB) in a mouse model, a drug repurposing study revealed. Data showed that the antiviral, a treatment for the hepatitis C virus (HCV) sold under the brand name Daklinza, extended RDEB mouse survival…

The enzyme polo-like kinase 1 (PLK1) may be a potential therapeutic target for squamous cell carcinoma (SCC) associated with recessive dystrophic epidermolysis bullosa (RDEB), according to an analysis of tumor tissue from an RDEB patient with recurring cancer. The gene that encodes PLK1 was almost seven times more active…