Marisa Wexler, MS,  senior science writer—

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

InMed Pharmaceuticals has submitted applications to regulatory authorities in Austria, Israel, and Serbia, seeking clearance to begin clinical testing of its investigational cannabinol cream INM-755 in people with epidermolysis bullosa (EB). Similar applications to authorities in France, Germany, Greece, and Italy are expected in the coming weeks.

INM-755, an investigational cannabinol cream for epidermolysis bullosa (EB), showed a positive safety profile and did not interfere with normal healing in a Phase 1 clinical trial conducted in healthy volunteers with open wounds, according to a press release from the cream’s developer, InMed Pharmaceuticals. INM-755…

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 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…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation and fast track status to AGLE-102, a potential therapy for dystrophic epidermolysis bullosa (DEB) being developed by Aegle Therapeutics. Rare pediatric disease designations are awarded investigative treatments for serious or life-threatening…

Filsuvez, a topical formulation being developed by Amryt Pharma, significantly improved wound closure in people with epidermolysis bullosa (EB) taking part in a Phase 3 clinical trial. According to Amryt, EASE (NCT03068780) is the first Phase 3 study to show a statistically significant increase in speed…

The combination of the oral antidepressant mirtazapine with a topical gel containing ketamine and amitriptyline successfully treated excessive itching in a man with epidermolysis bullosa pruriginosa (EBP). That case was described in the report “Epidermolysis Bullosa Pruriginosa successfully treated with concomitant topical and systemic…

The topical cream SD-101 failed to improve wound healing in a Phase 3 trial in people with epidermolysis bullosa (EB), a study reports. The study, “Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled…

A new collaboration between the University Carlos III Madrid (UC3M), Almirall, and the Medina Foundation aims to find treatments for recessive dystrophic epidermolysis bullosa (RDEB) and other genetic diseases caused by nonsense mutations, or those that result in shortened proteins. The project is partially funded by…

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…