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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…
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,…
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