News

A gene-edited skin cell therapy — called Spray-On Skin cells — promotes skin healing by correcting the mutation associated with recessive dystrophic epidermolysis bullosa (DEB), preclinical research from Avita Medical shows. “These data, while early, demonstrate promise … for treatment of epidermolysis bullosa with gene-corrected skin cells,” Mike Perry,…

The “Venture into Cures” virtual event raised more than $2.4 million to benefit the EB Research Partnership (EBRP), an organization dedicated to funding research into treatments and ultimately a cure for epidermolysis bullosa (EB). This second annual event, hosted by actor Tom Holland on Nov. 18, featured…

Treatment with Vyjuvek (previously called B-VEC) — a topical gene therapy for dystrophic epidermolysis bullosa (DEB) — improved wound healing with good tolerability over six months, according to top-line results of the Phase 3 GEM-3 trial. “We are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEK…

The U.S. Food and Drug Administration (FDA) has extended by three months its review of Amryt Pharma’s application seeking approval of Filsuvez (Oleogel-S10), a topical gel to treat skin wounds in people epidermolysis bullosa (EB). A regulatory decision is now expected on Feb. 28 — it previously was set…

Actor Tom Holland of “Spiderman” fame is hosting a live virtual event, with celebrity appearances and performances, to raise money to benefit the EB Research Partnership, which funds research for a cure and aims to increase awareness of epidermolysis bullosa (EB), according to a press release. Eddie…

Omalizumab — an antibody against immunoglobulin E (IgE) — may be effective and safe for wound healing in patients with recessive dystrophic epidermolysis bullosa (RDEB) and high IgE levels, a small study suggests. The treatment was also safe and well-tolerated, suggesting it may provide a therapeutic alternative for a disease…

Topline data should be available toward the end of this year for a Phase 3 clinical trial testing B-VEC (beremagene geperpavec), a topical gene therapy for treating skin wounds in patients with dystrophic epidermolysis bullosa (DEB). Krystal Biotech, the therapy’s developer, announced that the last of the 31 participants in…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

The U.S. Food and Drug Administration (FDA) has awarded Castle Creek Biosciences a research grant to support its Phase 3 clinical trial of FCX-007 (dabocemagene autoficel), an investigational gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). The grant of $1.82 million, which comes from…

From sharing stories to spreading the word about epidermolysis bullosa (EB) in person and on social media, supporters are poised to mark National Epidermolysis Bullosa Awareness Week, which runs annually in the U.S. from Oct. 25–31. According to the Dystrophic Epidermolysis Bullosa Research Association of America — coined as…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…