News

The European Medicines Agency (EMA) granted orphan drug designation to an investigational, and yet unnamed, potential therapy for recessive dystrophic epidermolysis bullosa (RDEB) based on CRISPR-Cas9 gene editing. This designation confers certain incentives, including advice from the EMA during the development process, reduced or exempted fees and…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

Krystal Biotech is planning to start a Phase 3 trial of B-VEC, a gene therapy candidate for the treatment of dystrophic epidermolysis bullosa (DEB), by June this year.  “We are pleased to report that our manufacturing batch…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tetra Bio-Pharma’s investigational cannabinoid cream PPP004 (THC-CBD) for the treatment of epidermolysis bullosa, the company has announced. Orphan drug status aims to encourage the development of therapies for rare diseases (those affecting less than 200,000 patients…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

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…