The U.S. Food and Drug Administration (FDA) has granted orphan drug status to APR-TD011, a spray therapy candidate for the treatment of epidermolysis bullosa (EB). This designation is intended to encourage the development of therapies for rare and serious diseases, which are expected to outperform existing alternatives. Among its…
After its recent clinical hold by regulators, the Phase 3 VIITAL trial has been given the green light to investigate EB-101, a gene-corrected cell therapy for people with recessive dystrophic epidermolysis bullosa (RDEB). Abeona Therapeutics, the company developing EB-101, plans to start VIITAL in early 2020. RDEB…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
Scientists have discovered a new mutation in the COL7A1 gene — which provides instructions for making a type of collagen protein — in a Chinese family with pretibial dystrophic epidermolysis bullosa (DEB), a study revealed. The study, “A novel mutation of COL7A1 in a Chinese DEB‐Pt family…
Taking multiple precautions can ensure that pregnancy, delivery, and the postpartum period are uncomplicated in women with epidermolysis bullosa (EB) simplex, a case study reports. The research, “Management of epidermolysis bullosa simplex in pregnancy: A case report,” was published in the journal Case Reports in Women’s Health.
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
InMed Pharmaceuticals has requested approval to start a Phase 1 trial in the Netherlands to test the cannabinoid-based treatment candidate INM-755 for epidermolysis bullosa (EB). Pending approval, the company plans to enroll patients until the end of the year. INM-755 is a topical cream being developed to…
Topical treatment with B-VEC, formerly known as KB103, is safe and leads to complete and durable wound healing in people with recessive dystrophic epidermolysis bullosa (RDEB), final results from a small Phase 1/2 trial suggest. RDEB is caused by mutations in the COL7A1 gene. This leads to…
The experimental cell therapy EB-101 was safe and led to successful wound healing in seven adults with recessive dystrophic epidermolysis bullosa (RDEB), followed-up for five years, the results of a Phase 1/2a clinical trial show. The study detailing the results, “Phase 1/2a clinical trial of gene-corrected…
The investigational oral treatment Serlopitant may be able to reduce itching in people with epidermolysis bullosa (EB), results from a small Phase 2 clinical trial suggest. However, most results did not achieve statistical significance, which means further studies will be needed to draw firm conclusions. The results were…
New guidelines on foot care for people with epidermolysis bullosa (EB) highlight the benefits of proper care and footwear, and recommend that a podiatrist be involved in routine health checks for people with EB. The guidelines, “Foot care in Epidermolysis bullosa: Evidence-based Guideline,” were published in the …
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