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…
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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,”…
In interviews, people with epidermolysis bullosa (EB) reported that living with these rare skin diseases has broad impacts on their psychosocial health. This burden was seen across physical, emotional, social, and functional domains, per the study, conducted in Europe. According to the researchers, the findings emphasize the importance of…
Children with recessive dystrophic epidermolysis bullosa (RDEB) have lower-than-normal levels of hormones that are important for growth, likely due to chronic inflammation, a study found. The data suggest that chronic inflammation in these children may lead to low levels of growth-driving hormones, ultimately resulting in poor growth. If…
A gene-editing therapy made to correct a genetic defect in the LAMB3 gene that’s known to cause junctional epidermolysis bullosa (JEB) worked as intended and restored more normal laminin 332 protein levels in skin cells from a JEB patient. To make the treatment more precise and safer, the scientists…
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, “…
An arm of the European Medicines Agency (EMA) is expected to issue its opinion early next year on whether to approve beremagene geperpavec-svdt, which is marketed as Vyjuvek in the U.S., for dystrophic epidermolysis bullosa (DEB). The Committee for Medicinal Products for Human Use (CHMP) has asked Krystal…
Surgical correction followed by continuous compressive bandaging effectively treated hand finger adhesion and contractures in a woman with recessive dystrophic epidermolysis bullosa (RDEB), according to a report. RDEB patients commonly undergo hand surgeries to improve function, and may require orthoses post-surgery to preserve results. Patients usually need several procedures.
RLF-TD011, a spray of hypochlorous acid that Relief Therapeutics is developing to control infections in wounds of epidermolysis bullosa patients, was effective at fighting off harmful bacteria in adults and children with the junctional or dystrophic form of the disease. That’s according to findings from an investigator-initiated…
The U.S. Food and Drug Administration (FDA) is expected to decide whether or not to approve the cell therapy pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB) by April 29, 2025. Abeona Therapeutics, the developer of pz-cel, announced that the FDA has agreed to review a…
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