Abeona Therapeutics is preparing for the potential launch of pz-cel (prademagene zamikeracel) to treat recessive dystrophic epidermolysis bullosa (RDEB), after the U.S. Food and Drug Administration (FDA) initiated discussions on the medication label and post-marketing requirements, according to a company press release. Last fall, the…
People with epidermolysis bullosa (EB) experience a deterioration in oral health, which is linked to poor health-related quality of life. That’s according to a study in Germany, which also found patients with dystrophic EB had poorer oral health and worse oral health-related quality of life when compared with…
A committee of the European Medicines Agency is recommending approving Vyjuvek (beremagene geperpavec), a gene therapy from Krystal Biotech, to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). The opinion by the Committee for Medicinal Products for Human Use (CHMP) will now go to…
Castle Creek Biosciences has secured $75 million in financing to support a Phase 3 trial testing its gene therapy to promote wound healing in people with dystrophic epidermolysis bullosa (DEB). The royalty financing agreement for Castle Creek‘s treatment, D-Fi, was led by Ligand Pharmaceuticals, which made…
France-based pharma company Theravia has obtained rights to an experimental molecule that may be used to treat epidermolysis bullosa (EB). The molecule, known as DCN-tCRK, was discovered by scientists at Tampere University, in Finland. Under the new licensing agreement, Theravia will be in charge of further drug development…
The stem cell therapy CORDStrom, in the pipeline at Inmune Bio, was found in a clinical trial to reduce itch and pain in children — from infants to teenagers — with intermediate and severe recessive dystrophic epidermolysis bullosa (RDEB), new data show. The treatment candidate also reduced disease…
A topical ointment containing diacerein, an arthritis medication, promoted wound healing in a 5-year-old girl with hard-to-treat recessive dystrophic epidermolysis bullosa (DEB), a case study reports. “These data characterize diacerein as a potential candidate for improving wound healing in RDEB through its impact on inflammatory [cells],” researchers wrote. The…
Adults with recessive dystrophic epidermolysis bullosa (RDEB) have abnormal immune cell profiles in blood and skin samples, a study reveals. The patients also had high blood levels of pro-inflammatory fat-like lipids and immune signaling proteins. These findings “underscore the concept of RDEB as [a] genetic disorder with distressed immunometabolism/inflammation,”…
In interviews, people with epidermolysis bullosa (EB) reported that living with these rare skin diseases has broad impacts on their psychosocial health. This burden was seen across physical, emotional, social, and functional domains, per the study, conducted in Europe. According to the researchers, the findings emphasize the importance of…
A gene-editing therapy made to correct a genetic defect in the LAMB3 gene that’s known to cause junctional epidermolysis bullosa (JEB) worked as intended and restored more normal laminin 332 protein levels in skin cells from a JEB patient. To make the treatment more precise and safer, the scientists…
Becoming a self-taught provider of home-based skin care is one of the many challenges faced by parents of children with epidermolysis bullosa (EB) — most of whom reported often being the primary caregiver for their affected sons and daughters in an interview-based study from Norway. Other challenges identified in…
Treatment with losartan, a commonly used oral blood pressure medication, safely reduced the signs and symptoms of recessive dystrophic epidermolysis bullosa (RDEB) in children, according to the results of a Phase 1/2 clinical trial. The repurposed medication particularly benefited young patients with severe disease, and was well tolerated with…
Get regular updates to your inbox.