Author Archives: Inês Martins PhD

The immunotherapy Libtayo (cemiplimab-rwlc) may be a promising treatment for the skin cancers that sometimes appear in people with recessive dystrophic epidermolysis bullosa (RDEB) as a result of chronic wounds, a case report shows. The study, “Induced Remission of Metastatic Squamous Cell Carcinoma with an…

Transplanting skin grafts from healthy donors can be a safe and effective way of treating chronic wounds in people with recessive dystrophic epidermolysis bullosa, a small clinical trial shows. But to create immune tolerance and prevent the graft from being rejected, patients first need to receive a…

Krystal Biotech has launched a pivotal Phase 3 clinical trial to investigate the topical gene therapy B-VEC (beremagene geperpavec) as a treatment for skin wounds in people with dystrophic epidermolysis bullosa (DEB). The GEM-3 trial (NCT04491604) is already recruiting participants with DEB at four sites in…

Intravenous immunoglobulin (IVIG) may be a promising treatment for reducing inflammation in severe epidermolysis bullosa pruriginosa (EBP) — a rare type of dystrophic epidermolysis bullosa (DEB) marked by severe itching of the skin — that fails to respond to other therapies, a case report suggests. A 43-year-old…

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…

Abeona Therapeutics announced the opening of the Elisa Linton Center for Rare Disease Therapies, a commercial manufacturing facility to support the development of its gene and cell therapy candidates targeting rare diseases like recessive dystrophic epidermolysis bullosa (RDEB). Based in Cleveland, Ohio, the GMP facility will be able to manufacture…

The U.S. Food and Drug Administration recently granted fast-track designation to Krystal Biotech’s KB103 for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is caused by mutations in the COL7A1 gene, the gene that provides instructions to make type VII collagen (COL7). Collagen strengthens body tissues and is…

Krystal Biotech recently dosed the first patient in a Phase 1/2 clinical trial testing KB103, a topical gene therapy candidate for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a progressive and painful form of epidermolysis bullosa characterized by blistering of the skin. The blistering happens…

Krystal Biotech recently announced that the European Medicines Agency (EMA) has granted the company’s investigational therapy KB103 orphan medicinal product designation for the treatment of dystrophic epidermolysis bullosa (DEB). KB103 is the first investigational non-infectious viral vector (HSV-1) gene therapy for DEB to receive this designation in Europe. In…

Debra of America (Dystrophic Epidermolysis Bullosa Research Association of America) and the Pachyonychia Congenita Project (PC Project) will soon meet with federal regulators to discuss patient-focused drug development in both rare skin diseases: epidermolysis bullosa (EB) and pachyonychia congenita (PC). PC is a rare genetic skin disorder caused by…

Hollywood celebrities Kaley Cuoco, Mandy Moore and Elizabeth Olsen have designed the latest Charity by Design collection by eco-conscious jewelry brand Alex and Ani to benefit the Epidermolysis Bullosa Medical Research Foundation (EBMRF). To support the epidermolysis bullosa (EB) community, Cuoco, Moore and Olsen were each responsible for the design…

Moin Younis, the ambassador for Acorns Children’s Hospice, won a Pride of Britain award as a “Teenager of Courage” for his character as a leader and for inspiring other children around him to stay strong despite life-limiting conditions. As the ambassador for Acorns, a center for children with life-limiting or life-threatening…