Researchers at four centers in France will evaluate the safety and effectiveness of apremilast, an approved oral psoriasis therapy, in children and adults with severe epidermolysis bullosa simplex (EBS) in an open-label Phase 2 clinical trial. Dubbed EBULO (NCT06509984), the study will test apremilast — currently sold as…
Scientists traced a common genetic mutation that causes recessive dystrophic epidermolysis bullosa (RDEB) to the Sephardic Jewish population, a group that has roots in Spain and Portugal. Even so, analysis showed that the RDEB patients who carry the mutation assessed in the study, both in Europe and the Americas, did…
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…
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