epidermolysis bullosa

[Onconova Therapeutics, Inc. (GLOBE NEWSWIRE)] Onconova Therapeutics, Inc., announced that the first patient has been dosed in an investigator-initiated Phase II study to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa-associated locally advanced/metastatic squamous cell carcinoma.

[JCI Insight] In a Phase I/IIa trial, scientists evaluated the safety and possible clinical efficacy of intravenous infusion of allogeneic human umbilical cord blood–derived mesenchymal stem cells in patients with recessive dystrophic epidermolysis bullosa.

[Abeona Therapeutics Inc.] Abeona Therapeutics Inc. held a successful Type B meeting with the US FDA to align with the Agency on the company’s proposal regarding co-primary endpoints for the pivotal Phase III VIITAL™ study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder where those inflicted have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin.

[Scientific Reports] Scientists used epithelial cells producing mutant keratin itermediate filament polypeptides that have been identified as the cause of the hereditary blister-forming skin disease epidermolysis bullosa simplex.

[Abeona Therapeutics, Inc.] Abeona Therapeutics, Inc. announced that the company held a successful Type B meeting with the FDA to align with the agency on the company’s proposal regarding co-primary endpoints for the pivotal Phase 3 VIITAL™ study of EB-101 in RDEB.

[Wound Repair and Regeneration] The authors investigated the therapeutic effects of allogeneic platelet gel prepared from umbilical cord blood in a group of children diagnosed with dystrophic epidermolysis bullosa eligible for surgical correction of pseudosyndactyly in the hand.