News

April 16, 2021April 16, 2021

Phase 3 Trial of B-VEC, Topical Gene Therapy for DEB, Fully Enrolled

Krystal Biotech announced the full enrollment of a pivotal Phase 3 clinical trial of its topical gene therapy B-VEC (beremagene geperpavec) as a skin wound treatment for people with dystrophic epidermolysis bullosa (DEB). ... Read more

April 15, 2021April 15, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

April 9, 2021April 9, 2021

Amryt Pharma Advances Regulatory Applications for Filsuvez

News

Amryt Pharma has completed its rolling submission of an application to the U.S. Food and Drug Administration seeking the approval of Filsuvez (Oleogel-S10), a topical gel to heal skin wounds in people with junctional (JEB) ... Read more

April 2, 2021April 2, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 26, 2021March 26, 2021

Stem Cells Derived From Skin Show Early Potential to Treat RDEB

News

Skin-derived stem cells producing the ABCB5 protein outperformed those derived from bone marrow at migrating toward and integrating into wounds associated with recessive dystrophic epidermolysis bullosa (RDEB), a study reports. ... Read more

March 19, 2021March 19, 2021

Patient-derived Skin Graft Found to Treat RDEB in Boy, 13

News

A 13-year-old boy with recessive dystrophic epidermolysis bullosa (RDEB) had his skin lesions eased after surgeons transplanted a skin graft grown from his back, according to a case study report. ... Read more

March 12, 2021March 12, 2021

4th Annual ‘Plunge for Elodie’ Goes Virtual to Raise Funds for EB Research

News

This month’s Plunge for Elodie will be a virtual event for a first time, with participants worldwide encouraged to plunge into freezing waters at their respective locations to raise funds in ... Read more

March 9, 2021March 9, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

News, syndicated

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 3, 2021March 3, 2021

Certain Coping Strategies May Help Boost Life Quality for Young Patients, Parents

News

Children with epidermolysis bullosa (EB) who accept their condition and emotionally detach themselves from it tend to have a better quality of life than children who are very emotional and ... Read more

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

News, syndicated

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more