News

The experimental cell therapy EB-101 was safe and led to successful wound healing in seven adults with recessive dystrophic epidermolysis bullosa (RDEB), followed-up for five years, the results of a Phase 1/2a clinical trial show. The study detailing the results, “Phase 1/2a clinical trial of gene-corrected…

The investigational oral treatment Serlopitant may be able to reduce itching in people with epidermolysis bullosa (EB), results from a small Phase 2 clinical trial suggest. However, most results did not achieve statistical significance, which means further studies will be needed to draw firm conclusions. The results were…

New guidelines on foot care for people with epidermolysis bullosa (EB) highlight the benefits of proper care and footwear, and recommend that a podiatrist be involved in routine health checks for people with EB. The guidelines, “Foot care in Epidermolysis bullosa: Evidence-based Guideline,” were published in the …

The U.S. Food and Drug Administration (FDA) has refused to approve the initiation of Abeona Therapeutics‘s Phase 3 clinical trial evaluating its gene-corrected cell therapy EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). The clinical hold, the company explained, is…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

A novel gene editing technique was able to correct mutations in COL7A1 in cells taken from people with recessive dystrophic epidermolysis bullosa (RDEB), providing a proof of concept for using the editing technique in the disease. The finding was published in the Journal of Investigative Dermatology in a study titled,…

A type of wound dressing, called dialkylcarbamoylchloride (DAAC)‐coated cotton acetate, closes infected wounds related to epidermolysis bullosa (EB) faster than the standard saline dressing plus a topic antibiotic, and with similar antibacterial effects, a study found. The study, “Clinical efficacy of dialkyl-carbamoyl chloride‐coated cotton acetate dressing…

Cell therapy with genetically engineered skin grafts requires that collagen type VII be “corrected” in two types of skin cells — keratinocytes and fibroblasts — to be effective against recessive dystrophic epidermolysis bullosa (RDEB), a study in mice has found. Researchers saw that only skin models made of collagen…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

RegeneRx Biopharmaceuticals announced the first patient enrolled in a Phase 2 clinical trial of RGN-137, the company’s investigational topical gel for treating epidermolysis bullosa (EB), achieved complete wound healing. RGN-137 is a gel form medication intended to accelerate wound healing when applied directly on skin lesions. It is…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Injection of mesenchymal stromal cells (MSCs) — cells that can be isolated from several tissues and may grow into different cell types when cultured in a lab dish — may prevent localized skin blistering sometimes seen in recessive dystrophic epidermolysis bullosa (RDEB), a study says. Results from the study,…