News

December 11, 2019December 11, 2019

FDA Clears Phase 3 Trial Testing EB-101 Cell Therapy for Severe Blistering Skin Disorder

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 ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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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 ... Read more

December 6, 2019December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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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 ... Read more

December 4, 2019December 4, 2019

New COL7A1 Gene Mutation Found in Rare DEB Subtype, Study Shows

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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 ... Read more

November 27, 2019November 27, 2019

Precautions Can Help Ensure Safe Pregnancy, Delivery in Women With EB Simplex, Study Reports

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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 ... Read more

November 13, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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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. ... Read more

November 13, 2019November 13, 2019

Cannabinoid-based INM-755 May Soon be Tested in Phase 1 Trial in Netherlands

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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 ... Read more

November 6, 2019November 6, 2019

Gene Therapy B-VEC Is Safe, Leads to Sustained Wound Healing in RDEB Patients, Phase 1/2 Trial Shows

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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 ... Read more

October 30, 2019October 30, 2019

EB-101 Cell Therapy Safely Heals Wounds in Adults with RDEB, Early Trial Results Show

News

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 ... Read more

October 18, 2019

Serlopitant May Reduce Itching in EB, But More Research Is Needed

News

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 ... Read more

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