News

May 6, 2020May 6, 2020

Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June

Krystal Biotech is planning to start a Phase 3 trial of B-VEC, a gene therapy candidate for the treatment of dystrophic epidermolysis bullosa (DEB), by June this year. “We ... Read more

April 29, 2020April 29, 2020

Cannabinoid Cream PPP004 Receives FDA Orphan Drug Designation to Treat EB

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tetra Bio-Pharma’s investigational cannabinoid cream PPP004 (THC-CBD) for the treatment of epidermolysis bullosa, the company has announced. Orphan ... Read more

April 27, 2020April 27, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

News, syndicated

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in ... Read more

April 24, 2020April 24, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

News, syndicated

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. ... Read more

April 22, 2020April 22, 2020

Oral Dapsone Can Lead to Rapid, Sustained Improvement in Inherited EB, Case Report Shows

News

Oral treatment with dapsone, an antibiotic and anti-inflammatory, may be a valuable therapy for patients with inherited epidermolysis bullosa, according to a case report that showed the therapy resulted ... Read more

April 15, 2020April 15, 2020

Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle

News

SkinGun, a device designed to gently spray stem cells onto the skin of severe burn victims, is being tested in an early study of a potential gene therapy for ... Read more

April 9, 2020April 9, 2020

Phase 1 Trial of EB Cannabinol Cream Completes Treatment in Healthy Volunteers

News

All healthy volunteers participating in InMed Pharmaceuticals’ Phase 1 trial of INM-755, an investigational cannabinol cream for epidermolysis bullosa (EB), have completed treatment and clinical evaluation. InMed expects to ... Read more

April 3, 2020April 3, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most ... Read more

April 2, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

News, syndicated

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most ... Read more

April 1, 2020April 1, 2020

$75M Funding to Advance Gene Therapy for RDEB Entering Phase 3 Trial

News

Castle Creek Biosciences has received a $75 million investment to advance its gene therapy program, including FCX-007, a treatment candidate for people with recessive dystrophic epidermolysis bullosa (RDEB) that now ... Read more

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