News

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

November 18, 2020November 18, 2020

Eddie Vedder Hosts ‘Venture Into Cures,’ Virtual Event Supporting EB Research

News

Tonight at 8 p.m. EST, Jill and Eddie Vedder will host Venture Into Cures, a virtual event to raise awareness of epidermolysis bullosa (EB), and also raise funds through ... Read more

November 11, 2020November 11, 2020

1st Patient Treated in Phase 3 Trial of RDEB Gene Therapy, D-Fi

News

A first patient has been treated in a Phase 3 trial testing the investigational gene therapy D-Fi (debcoemagene autoficel), formerly known as FCX-007, on wounds in people with recessive ... Read more

November 5, 2020November 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 4, 2020November 4, 2020

Filsuvez Raises Patients’ Chance of Complete and Quick Wound Healing, Trial Finds

News

Filsuvez, a topical gel by Amryt Pharma, raises the probability of complete wound closure while reducing the time that wounds take to heal in people with epidermolysis bullosa (EB), new data ... Read more

October 28, 2020October 28, 2020

1st RDEB Patient Dosed With PTR-01 Protein Replacement Therapy in Phase 2 Trial

News

A Phase 2 trial of PTR-01 (also known as BBP-589) as a treatment for recessive dystrophic epidermolysis bullosa (RDEB) has dosed its first patient. The study, known as PTR-01-002 (NCT04599881), is ... Read more

October 27, 2020October 28, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

News, syndicated

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more

October 21, 2020October 21, 2020

Debra Plans Events in US, UK to Mark EB Awareness Week

News

The last week in October is Epidermolysis Bullosa Awareness Week, and Debra branches in both the U.S. and the U.K. have events and initiatives planned to support the patient community. ... Read more

October 20, 2020October 20, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more

October 14, 2020October 14, 2020

Topical Anesthetic May Ease Pain of Dressing Changes With Severe EB, Trial Suggests

News

Topical ropivacaine, a local anesthetic marketed as Naropin, eased the pain associated with wound care in children with severe epidermolysis bullosa (EB) in a small clinical trial. The study, “Efficacy ... Read more