News

January 22, 2020January 22, 2020

Abeona Launches Phase 3 Trial Testing EB-101 Cell Therapy for RDEB

Abeona Therapeutics has begun its Phase 3 VIITAL trial evaluating EB-101, a gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the company recently announced. An ... Read more

January 15, 2020January 15, 2020

Castle Creek Acquires Fibrocell, Now Leading Development of FCX-007 Gene Therapy for RDEB

News

Castle Creek Pharmaceutical recently acquired Fibrocell Science and is now leading the development of treatments for epidermolysis bullosa (EB), including the investigational gene therapy FCX-007 for the treatment of recessive ... Read more

January 8, 2020January 8, 2020

Research Consortium Receives $1.6M to Develop Stem Cell Therapies for Skin Diseases

News

The Epidermolysis Bullosa iPS Cell Consortium has received $1.6 million in funding to develop stem cell-based therapies for people with inherited skin diseases such as epidermolysis bullosa (EB). A grant ... Read more

December 18, 2019December 18, 2019

Spray Therapy APR-TD011 Earns FDA Orphan Drug Designation to Treat EB

News

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 ... Read more

December 11, 2019December 11, 2019

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

News

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

News

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

News

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

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