News

November 13, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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

News

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

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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

October 4, 2019

New Guidelines for Foot Care in EB Stress Use of Podiatrists, Proper Footwear

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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 ... Read more

September 27, 2019

FDA Halts Phase 3 Trial of Abeona’s EB-101 for RDEB Due to Transport Concerns

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The U.S. Food and Drug Administration (FDA) has refused to approve the initiation of Abeona Therapeutics‘s Phase 3 clinical trial evaluating its gene-corrected cell therapy EB-101 for recessive dystrophic epidermolysis ... Read more

September 27, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 20, 2019

New Gene Editing Technique Shown to Correct COL7A1 Gene in RDEB Cells

News

A novel gene editing technique was able to correct mutations in COL7A1 in cells taken from people with recessive dystrophic epidermolysis bullosa (RDEB), providing a proof of concept for using ... Read more

September 13, 2019

DAAC Dressing Closes EB Wounds Faster Than Standard Saline-Antibiotic One, Study Says

News

A type of wound dressing, called dialkylcarbamoylchloride (DAAC)‐coated cotton acetate, closes infected wounds related to epidermolysis bullosa (EB) faster than the standard saline dressing plus a topic antibiotic, and ... Read more

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