News

October 14, 2020October 14, 2020

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

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

October 8, 2020October 8, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

News, syndicated

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more

October 7, 2020October 7, 2020

AGLE-102 for DEB Given Rare Pediatric Disease, Fast Track Status by FDA

News

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation and fast track status to AGLE-102, a potential therapy for dystrophic epidermolysis bullosa (DEB) being developed ... Read more

September 30, 2020September 30, 2020

Orphan Drug Status in Europe Likely for AP103, DEB Gene Therapy

News

A potential gene therapy for dystrophic epidermolysis bullosa (DEB) known as AP103 received a positive opinion from the Committee for Orphan Medicinal Products (COMP) — an arm of the ... Read more

September 23, 2020September 23, 2020

Amryt Plans Regulatory Submissions for Filsuvez Following Positive Phase 3 Data

News

Filsuvez, a topical formulation being developed by Amryt Pharma, significantly improved wound closure in people with epidermolysis bullosa (EB) taking part in a Phase 3 clinical trial. According to ... Read more

September 16, 2020September 22, 2020

Libtayo May Treat Advanced Skin Cancer in RDEB Patients, Case Report Shows

News

The immunotherapy Libtayo (cemiplimab-rwlc) may be a promising treatment for the skin cancers that sometimes appear in people with recessive dystrophic epidermolysis bullosa (RDEB) as a result of chronic ... Read more

September 9, 2020September 9, 2020

Donor-derived Skin Grafts Feasible Way of Treating RDEB, Trial Reports

News

Transplanting skin grafts from healthy donors can be a safe and effective way of treating chronic wounds in people with recessive dystrophic epidermolysis bullosa, a small clinical trial shows. ... Read more

September 3, 2020September 3, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

September 2, 2020September 2, 2020

Efficacy of Cream in Control Group May Explain ESSENCE Trial Failure, Study Reports

News

An analysis of the control group from the ESSENCE Phase 3 clinical trial, which tested the topical cream SD-101 in people with epidermolysis bullosa (EB), found overall improvements in wound closure and ... Read more

August 26, 2020August 26, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News, syndicated

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

Pin It on Pinterest