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Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.
When applied directly over skin wounds, the antibiotic gentamicin can restore the production of laminin 332 and promote wound closure in people with generalized severe junctional epidermolysis bullosa (GS-JEB), a Phase 1/2 trial shows. The findings were reported in the study “Gentamicin Induces Laminin…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
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…
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