News

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…

Pain is a frequent problem for people with recessive dystrophic epidermolysis bullosa (RDEB), and available medications are often not sufficient to control it, a study reported. “Pain is an almost universal symptom across all types of RDEB,” the researchers wrote, noting that worse pain “generally correlates with worse quality…

RLF-TD011, an antimicrobial spray, will be granted European Union patent coverage for wound treatment in people with epidermolysis bullosa (EB). The Relief Therapeutics treatment contains highly pure hypochlorous acid, a chemical with antimicrobial and anti-inflammatory properties. RLF-TD011 is marketed as Nexodyn for the management of chronic and acute…

Treatment with SNT-6935, an experimental compound that acts to reduce the activity of an enzyme that breaks down proteins, reduced skin damage in a lab model of epidermolysis bullosa acquisita (EBA), a study showed. The study, “Granzyme B inhibition reduces autoantibody-induced dermal–epidermal separation in an ex…

Three types of molecules were found to support the production of collagen VII (C7) in patient-derived cell models of recessive dystrophic epidermolysis bullosa (RDEB), according to a recent study. These compounds acted in RDEB caused by premature termination codon (PTC) mutations that produce a shorter, non-functional version of C7,…

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]…

Treatment with Janus kinase (JAK) inhibitors — medications such as upadacitinib and baricitinib — was seen to work better than dupilumab at easing itching and skin lesions in people with epidermolysis bullosa pruriginosa (EBP) in a small real-world study. Specifically, among five patients with known itch scores receiving dupilumab,…

Treatment with dupilumab, an injection therapy approved for certain inflammatory disorders, was found to completely heal blistering in a woman in Germany with epidermolysis bullosa acquisita (EBA), a case report describes. The woman’s healing with dupilumab came after “different systemic treatments … remained ineffective,” the researchers wrote. Indeed, the patient…

Children with dystrophic epidermolysis bullosa (DEB) in the U.S. are more likely to have visits to the emergency department during summertime compared with kids who don’t have the rare disease, according to a new study. Conversely, that dynamic was found to be reversed in the autumn months, with fewer…

Scientists have devised a new, more efficient method to create gene-edited stem cell therapies for dystrophic epidermolysis bullosa (DEB). “Solving critical bottlenecks, we refine a practical and simplified … protocol for the generation of genetically corrected … skin grafts … for the long-term healing of DEB patient wounds,” the…

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…