May 6, 2020May 6, 2020
Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June
News
Krystal Biotech is planning to start a Phase 3 trial of B-VEC, a gene therapy candidate for the treatment ... Read more
April 30, 2020April 30, 2020
NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts
syndicated
As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases ... Read more
April 29, 2020April 29, 2020
Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak
Announcements
FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are joining forces to provide patients ... 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 ... 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 ... 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 ... 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, ... 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 ... 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 ... 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 ... 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 ... 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 ... Read more

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 30, 2020April 30, 2020

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

syndicated

As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases — whose immune systems were generally compromised even before the ... Read more

April 29, 2020April 29, 2020

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

Announcements

FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and ... 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