News

Oral treatment with dapsone, an antibiotic and anti-inflammatory, may be a valuable therapy for patients with inherited epidermolysis bullosa, according to a case report that showed the therapy resulted in rapid and sustained benefits in two men with the disorder. The study, “Successful dapsone therapy in…

SkinGun, a device designed to gently spray stem cells onto the skin of severe burn victims, is being tested in an early study of a potential gene therapy for people with recessive dystrophic epidermolysis bullosa (RDEB). The preclinical work, underway at King’s College…

All healthy volunteers participating in InMed Pharmaceuticals’ Phase 1 trial of INM-755, an investigational cannabinol cream for epidermolysis bullosa (EB), have completed treatment and clinical evaluation. InMed expects to report final results of the trial, which was conducted in The Netherlands, later this year. The announcement came after…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Castle Creek Biosciences has received a $75 million investment to advance its gene therapy program, including FCX-007, a treatment candidate for people with recessive dystrophic epidermolysis bullosa (RDEB) that now in a pivotal clinical trial. “We are proud to have the strategic support of world-class investors whose impact…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

InMed Pharmaceuticals does not expect that the COVID-19 pandemic to significantly affect data collection in its Phase 1 trial testing INM-755, a potential epidermolysis bullosa (EB) treatment, in healthy volunteers. INM-755 is a topical cream, whose active ingredient is cannabinol (CBN), a compound present in trace amounts…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

The first patient has been treated in the VIITAL Phase 3 trial testing EB-101, a gene-corrected cell therapy candidate for people with recessive dystrophic epidermolysis bullosa (RDEB). Abeona Therapeutics, the company developing EB-101, announced the launch of this trial earlier this year following clearance…

Family members caring for children with epidermolysis bullosa (EB) report considerable stress, fatigue and feelings of helplessness, and need greater social and financial support, a small Taiwanese study based on interviews with 10 caregivers reports. This need is particularly acute in Taiwan, its researchers note, because that country lacks…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…