January 22, 2020January 22, 2020
Abeona Launches Phase 3 Trial Testing EB-101 Cell Therapy for RDEB
News
Abeona Therapeutics has begun its Phase 3 VIITAL trial evaluating EB-101, a gene-corrected cell therapy for the treatment of ... Read more
January 21, 2020January 17, 2020
It’s My Body: Why Unsolicited Comments Can Be Hurtful
Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl
It’s annoying — yes, freaking annoying — when strangers and even acquaintances feel compelled to comment on another person’s ... 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), ... 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 ... 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 ... Read more
December 17, 2019December 13, 2019
All I Want for Christmas Is Everything I Already Have
Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl
When thinking of a topic for this column, I considered writing on a winter theme. But then, when I ... 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 ... Read more
December 10, 2019December 10, 2019
Gene Therapy Takes Center Stage at 2019 NORD Summit
News
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of ... Read more
December 6, 2019December 6, 2019
HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
News
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases ... Read more
December 4, 2019December 4, 2019
New COL7A1 Gene Mutation Found in Rare DEB Subtype, Study Shows
News
Scientists have discovered a new mutation in the COL7A1 gene — which provides instructions for making a type of ... 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 ... Read more
November 19, 2019November 15, 2019
I Look Forward to Telling My Epidermolysis Bullosa Story
Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl
I’ve been wanting to write about life with epidermolysis bullosa (EB) — my life as a so-called butterfly child ... Read more

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 21, 2020January 17, 2020

It’s My Body: Why Unsolicited Comments Can Be Hurtful

Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl

It’s annoying — yes, freaking annoying — when strangers and even acquaintances feel compelled to comment on another person’s appearance. I’ve heard comments like, “Your skin looks better than ... 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 17, 2019December 13, 2019

All I Want for Christmas Is Everything I Already Have

Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl

When thinking of a topic for this column, I considered writing on a winter theme. But then, when I think of December, Christmas comes to mind, rather than the ... 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

News

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

News

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

News

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