The first recessive dystrophic epidermolysis bullosa (RDEB) patient will be dosed with the cell-based gene therapy Zevaskyn (prademagene zamikeracel) in the next few months, with several others lined up to follow, according to developer Abeona Therapeutics. That patient has already been selected, and their skin cells have been…
A boy with junctional epidermolysis bullosa (JEB) given combined gene and stem cell treatment in 2015 was found six years later to have a distinctive bacterial population in healthy, blistered, and transplanted portions of his skin, according to a new study from researchers in Germany. Compared with healthy controls,…
A new six-minute film called “Living Between Pain and Hope” spotlights the impact that living with epidermolysis bullosa (EB) has on two people with the fragile skin condition. The film shares the experiences of a pair of young adults living on different continents but with the same rare…
A buildup of pro-inflammatory signaling molecules in skin wounds may explain the slow healing process experienced by people with recessive dystrophic epidermolysis bullosa (RDEB), a study suggested. The researchers said the findings support the development of therapies targeting wound-associated pro-inflammatory signals to prevent…
Lucile Packard Children’s Hospital Stanford, a medical facility in the San Francisco Bay Area, has completed the requirements needed to become a qualified treatment center for Zevaskyn (prademagene zamikeracel), a gene therapy approved earlier this year for recessive dystrophic epidermolysis bullosa (RDEB), according to a press release. The…
PKC412, a medication already used for treating certain blood cancers, has the potential to be repurposed for treating epidermolysis bullosa simplex (EBS), according to a preclinical study. Using skin cells from people with various EBS-causing genetic mutations, scientists found that PKC412, also known as midostaurin, made the cells more…
The first person has been dosed in a Phase 3 trial testing KB803, Krystal Biotech’s experimental eye drop formulation of the gene therapy beremagene geperpavec (B-VEC), for the treatment of eye problems in people with dystrophic epidermolysis bullosa (DEB). Krystal already markets a topical gel formulation of B-VEC…
Treatment with dupilumab, a medicine approved for certain inflammatory disorders, eases itch and disease severity in people with different subtypes of dystrophic epidermolysis bullosa (DEB), a small study suggests. Dupilumab-related benefits seen in a woman before she stopped treatment and got pregnant were mostly maintained throughout her pregnancy and…
Long-term treatment with the topical gene therapy Vyjuvek (beremagene geperpavec) was associated with continued wound closure and high patient satisfaction among people with dystrophic epidermolysis bullosa (DEB) who took part in an open-label extension (OLE) study. The therapy was also well tolerated over up to more than two…
Traws Pharma said it will advance development of skin cancer therapy rigosertib, after data showed the therapy led to a partial or complete treatment response for four recessive dystrophic epidermolysis bullosa (RDEB) patients with advanced forms of the skin cancer squamous cell carcinoma (SCC). The data came from two…
Rheacell and AOP Health are partnering to make stem cell therapies available for people with epidermolysis bullosa (EB) in Europe and elsewhere. “This partnership marks a significant milestone for Rheacell and the further development of our stem cell medication programs,” Christoph Ganss, MD, Rheacell’s CEO, said in…
Researchers used a small molecule called M3814 for more accurate editing of genetic mutations causing recessive dystrophic epidermolysis bullosa (RDEB), according to a new study. This method enabled skin cells from three patients to restore their production of type VII collagen (C7) protein, which they lacked, the researchers noted.
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