News

Lena Riedl, 24, has had epidermolysis bullosa (EB) since birth. Despite the suffering, she’s managed to turn her genetic “butterfly disease” into a career while inspiring her father, Rainer Riedl, to launch the world’s first dedicated EB clinic in her native Austria. The cheerful young woman is a communications and…

Castle Creek Pharmaceuticals‘ investigational treatment CCP-020 (diacerein 1% ointment) was granted Fast Track designation by the U.S. Food and Drug Administration to treat a type of epidermolysis bullosa (EB) called EB simplex (EBS). Castle Creek is investigating CCP-020 to treat EBS patients in an ongoing Phase 2 trial…

RGN-137, an experimental therapy for wound healing in epidermolysis bullosa (EB), is expected to advance into a Phase 3 trial, backed by a joint venture created by RegeneRx licensee GtreeBNT and YuYang DNU…

Amryt Pharma’s investigational therapy AP101, designed to reduce the time it takes for skin wounds to close, was granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration for the treatment of children with epidermolysis bullosa (EB). Rare Pediatric Disease status is granted to…

Treatment targeting a specific molecule involved in inflammation — called PI3K-delta — eased skin lesions and prevented blistering in mouse models of epidermolysis bullosa acquisita (EBA), according to a new study. The research, “Therapeutic Effect of a Novel Phosphatidylinositol-3-Kinase δ Inhibitor in Experimental Epidermolysis Bullosa Acquisita,”…

The commonly used antibiotic gentamicin (brand name Garamycin) can reverse the effects of about 80% of genetic mutations affecting the LAMB3 gene that cause Herlitz junctional epidermolysis bullosa (H-JEB), according to researchers. This finding was reported in the study, “Gentamicin induces LAMB3 nonsense mutation readthrough and restores…

A gel made from platelets — tiny blood cells involved in blood clotting — collected from umbilical cord blood showed positive results for the topical treatment of patients with epidermolysis bullosa (EB), according to a pilot study. Results of the study, “Cord blood platelet gel for the treatment…

The 2018 Debra Care Conference — a U.S. gathering of epidermolysis bullosa (EB) patients, families, disease advocates, and experts — gets underway this Sunday in suburban Phoenix. The biennial event, organized by the Dystrophic Epidermolysis Bullosa Research Association of America (Debra), takes place July 22-25 at the Sheraton Grand at…

An enzyme called granzyme B (GzmB) may be a key mediator of blistering in patients with epidermolysis bullosa acquisita (EBA) and other autoimmune skin disorders, according to a Canadian study. These findings suggest that a single treatment strategy could be developed for different conditions. The research, “…

A case report shows that epidermolysis bullosa acquisita (EBA), a rare form of epidermolysis bullosa that usually occurs in adulthood, can appear at earlier ages, even during adolescence. The study highlights the importance of considering EBA when diagnosing younger patients with suspicious clinical findings.

A Phase 1/2 clinical trial assessing investigational QR-313 as a treatment for dystrophic epidermolysis bullosa (DEB) has begun and is now enrolling participants, ProQR Therapeutics recently announced. The randomized, double-blind, placebo-controlled WINGS trial is recruiting up to eight participants with recessive DEB — a more severe form of the disease —…

Castle Creek Pharmaceuticals recently enrolled its first patient in a Phase 1 clinical trial evaluating the pharmacokinetics (how the drug is processed in the body) and safety of a new formulation of diacerein 1% ointment (CCP-020) in patients with epidermolysis bullosa (EB). The study (NCT03472287) is…