News

The U.S. Food and Drug Administration (FDA) has granted priority review to an application seeking approval of pz-cel (prademagene zamikeracel) — a cell therapy previously known as EB-101 — for people with recessive dystrophic epidermolysis bullosa (RDEB). Granting priority review shortens the time it will take the agency to…

EB Research Partnership (EBRP), an organization dedicated to funding research into treatments for epidermolysis bullosa (EB), has raised more than $5 million through a three-day series of events in October. The outcome is “beyond what we could have ever imagined,” Michael Hund, CEO of EBRP, said in a…

Inflammation occurring early in the course of recessive dystrophic epidermolysis bullosa (RDEB) may create conditions for the disease to progress and for cancer to grow in the skin, a mouse study suggests. Although further studies are necessary, the findings “underscore the essential role of inflammation in RDEB pathophysiology [disease]…

Healiva and C4U will collaborate to develop a next-generation gene editing therapy — focusing on new technology to correct gene mutations — for epidermolysis bullosa (EB) patients, who are sometimes known as butterfly children for their fragile skin. Combining C4U’s expertise in gene editing technology and Healiva’s…

A young woman whose dystrophic epidermolysis bullosa (DEB) pruriginosa was caused by a newly identified gene mutation responded well to treatment with dupilumab, a medication that relieves itching, researchers in China report. Dupilumab, approved as Dupixent for several other diseases, is given as a subcutaneous or under-the-skin…

Rigosertib may hold promise for recessive dystrophic epidermolysis bullosa (RDEB) associated with advanced squamous cell carcinoma (SCC), with two patients achieving complete treatment responses in the skin, according to initial clinical trial data. The findings were presented at the European Academy of Dermatology and Venereology (EADV) annual congress,…

A new registry for those with epidermolysis bullosa (EB) and their families, from the patient advocacy organization debra of America, is aiming to help both them and scientists to better understand this group of rare skin disorders. The ultimate goal, according to the nonprofit, is to develop new…

The diabetes medication metformin slowed tumor growth and prolonged survival in a mouse model of aggressive squamous cell carcinoma (SCC), the most common type of cancer seen in people with recessive dystrophic epidermolysis bullosa (RDEB), in a recent study.  It did so by inhibiting cellular processes used by…

Abeona Therapeutics is seeking U.S. Food and Drug Administration (FDA) approval for EB-101, an investigational cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). The biologics license application (BLA) submitted by the company was filed along with a request for priority review, which, if granted, would shorten the…

A rare case of epidermolysis bullosa acquista (EBA) was found in an older man in France with systemic sclerosis (SSc), according to a new report by researchers who noted the unusual co-occurrence of the two autoimmune diseases. “Several autoimmune or inflammatory diseases have been reported in association with…

Eddie Vedder of the rock band Pearl Jam will perform two concerts to support EB Research Partnership (EBRP) following its inaugural Venture Into Cures Summit and Dinner, set for Oct. 22 in Seattle, Washington. The organization funds research into treatments for epidermolysis bullosa (EB), a group of…

Itch is a common symptom with recessive dystrophic epidermolysis bullosa (RDEB) and available treatments are generally inadequate for managing it. That’s according to “Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study,” published in the Orphanet Journal of Rare Diseases. “The…