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June 18, 2018June 18, 2018

Allogeneic Fibroblast Injections into Skin Lesions Are Safe, Effective for RDEB, Study Shows

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Allogeneic fibroblast injections are an effective and safe treatment strategy for non-healing wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB), new research from Russia shows. The study, “Allogeneic ... Read more

June 13, 2018June 13, 2018

ProQR to Receive Up to $5 Million from Nonprofits to Develop QR-313 for DEB

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ProQR Therapeutics is partnering with the nonprofit organizations EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF) to develop QR-313, a treatment candidate for dystrophic epidermolysis bullosa. Dystrophic epidermolysis bullosa ... Read more

June 6, 2018June 8, 2018

Serlopitant’s Potential to Manage Itch in EB Patients Shown in Exploratory Trial

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Menlo Therapeutics’ investigative therapy serlopitant seems to effectively reduce itch in patients with epidermolysis bullosa, according to results from a small exploratory Phase 2 trial. The trial results were presented ... Read more

June 4, 2018June 4, 2018

Abeona Opens Manufacturing Plant to Support EB-101, and Other Gene and Cell Therapy Candidates

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Abeona Therapeutics announced the opening of the Elisa Linton Center for Rare Disease Therapies, a commercial manufacturing facility to support the development of its gene and cell therapy candidates targeting ... Read more

June 4, 2018June 4, 2018

US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

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As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third ... Read more

May 30, 2018May 30, 2018

EB Treatment Candidate BPM 31510 Granted Orphan Drug Designation in US

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BERG’s BPM 31510 (ubidecarenone), a treatment candidate for patients with epidermolysis bullosa (EB), the company announced. ... Read more

May 30, 2018May 30, 2018

#ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says

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Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of ... Read more

May 29, 2018May 29, 2018

#ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

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Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different ... Read more

May 25, 2018May 25, 2018

FDA Grants Fast-Track Designation for Krystal Biotech’s Gene Therapy Candidate KB103 to Treat DEB

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The U.S. Food and Drug Administration recently granted fast-track designation to Krystal Biotech’s KB103 for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is caused by mutations in the ... Read more

May 23, 2018May 23, 2018

Positive Interim Results of Phase 1/2 Trial Support FCX-007 Gene Therapy for RDEB

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FCX-007, an experimental gene therapy developed by Fibrocell Science in collaboration with Precigen (a subsidiary of Intrexon), continues to improve wound healing in recessive dystrophic epidermolysis bullosa (RDEB) patients, new ... Read more

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