News

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…

People with epidermolysis bullosa (EB) often use topical skin care products and need to be aware that products containing antimicrobial agents, and bath water with high concentrations of cleansing additives, could carry risks, a study reported. The study, “Skin cleansing and topical product use in patients…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Using mesenchymal stem cells to treat adults with recessive dystrophic epidermolysis bullosa (RDEB) appears to be safe and may ease symptoms such as itching for at least two months, a Phase 1/2 clinical trial shows. The results were described in the study “A Phase I/II open-label…