November 19, 2020November 17, 2020
When EB Steals Your Cat
Columns, Fragile but Fierce – a Column by Patrice Williams
My mom got an adorable kitten last weekend. When I showed my boys the sweet picture she texted us, ... Read more
November 19, 2020November 19, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
News, syndicated
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how ... 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 ... 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), ... 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 ... Read more
November 5, 2020November 3, 2020
The Difficult Decision in Favor of a Feeding Tube, Part 2
Columns, Fragile but Fierce – a Column by Patrice Williams
Last in a series. Read part one. After our son Jonah was born with junctional epidermolysis bullosa, we made ... 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 ... 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 ... 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 ... Read more
October 27, 2020October 23, 2020
Say Hello to This Year’s Cover Model
Columns, Girl with the Butterfly Tattoo – a Column by Lena Riedl
This month, one of my childhood dreams came true. Ever since I was 15, I secretly wanted to be ... Read more
October 22, 2020October 20, 2020
The Difficult Decision in Favor of a Feeding Tube, Part 1
Columns, Fragile but Fierce – a Column by Patrice Williams
First in a two-part series. Our son Jonah, who was born with junctional epidermolysis bullosa in 2009, struggled to ... 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 ... Read more

My mom got an adorable kitten last weekend. When I showed my boys the sweet picture she texted us, Jonah questioned me about our cat that we re-homed when ... Read more

November 19, 2020November 19, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

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 5, 2020November 3, 2020

The Difficult Decision in Favor of a Feeding Tube, Part 2

Columns, Fragile but Fierce – a Column by Patrice Williams

Last in a series. Read part one. After our son Jonah was born with junctional epidermolysis bullosa, we made the difficult decision for him to get a gastrostomy feeding ... 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 27, 2020October 23, 2020

Say Hello to This Year’s Cover Model

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

This month, one of my childhood dreams came true. Ever since I was 15, I secretly wanted to be a model. But because I have scars on my body ... Read more

When EB Steals Your Cat