News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AP103, a potential topical gene therapy for dystrophic epidermolysis bullosa (DEB). “Receiving an orphan drug designation from the FDA for our gene-therapy candidate, AP103, is a significant development for patients suffering…

Epidermolysis bullosa (EB) may be more common in the Netherlands than previously assumed, a long-term Dutch study suggests. Results showed that EB now has a higher prevalence — the number of cases present in a particular population at a given time — than had been thought. Notably, EB simplex…

A gel made from platelets — tiny cells involved in blood clotting — that were collected from umbilical cord blood was better and faster at promoting wound healing in children with recessive dystrophic epidermolysis bullosa (RDEB) than a standard approach, a pilot study found. The children were treated with the…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

INM-755, an investigational cannabinol cream for epidermolysis bullosa (EB), showed a good safety profile in a Phase 1 clinical trial conducted in healthy volunteers, according to a press release from InMed Pharmaceuticals, which is developing the treatment. INM-755 is a topical cream containing cannabinol (CBN), a compound…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

A modified version of cannabidiol has potent antioxidant and anti-inflammatory properties that may help to treat epidermolysis bullosa (EB), research suggests. Cannabidiol, or CBD, is one of the most studied active chemicals — called cannabinoids — of the cannabis plant. CBD is devoid of the mind-altering effects…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Tonight at 8 p.m. EST, Jill and Eddie Vedder will host Venture Into Cures, a virtual event to raise awareness of epidermolysis bullosa (EB), and also raise funds through the stories of individuals and families living with the disorder and a series of celebrity performances and special appearances.

A first patient has been treated in a Phase 3 trial testing the investigational gene therapy D-Fi (debcoemagene autoficel), formerly known as FCX-007, on wounds in people with recessive dystrophic epidermolysis bullosa (RDEB). “Our late-stage Phase 3 trial of D-Fi continues to progress for the localized treatment…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…

Filsuvez, a topical gel by Amryt Pharma, raises the probability of complete wound closure while reducing the time that wounds take to heal in people with epidermolysis bullosa (EB), new data from a Phase 3 clinical trial show. Investigators found a statistically significant 41.3% of patients treated Filsuvez had…