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December 10, 2018December 10, 2018

Phase 3 Trial of RDEB Therapy EB-101 to Start in 2019, Abeona Therapeutics Says

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Abeona Therapeutics plans to start a Phase 3 trial in 2019 testing the cell therapy candidate EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB). The multi-center, randomized study, ... Read more

December 3, 2018December 3, 2018

TXA127 Enters Development Program as Potential Treatment for EB

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Constant Pharmaceuticals recently announced plans to begin a development program for TXA127 for the treatment of epidermolysis bullosa (EB). The potential therapy is expected to enter Phase 2 clinical ... Read more

November 26, 2018November 26, 2018

Diacerein Ointment Could Be Safe Treatment for Generalized Severe EBS, Study Suggests

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A 1% diacerein ointment could be a safe and well-tolerated way to treat generalized severe epidermolysis bullosa simplex (EBS), according to a study involving two boys treated for four weeks. In ... Read more

November 12, 2018November 12, 2018

Intravenous Immunoglobulin and Rituximab Most Effective EBA Treatments, Study Reports

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Treatment with intravenous immunoglobulin (IVIG) and off-label rituximab can lead to complete remission of epidermolysis bullosa acquisita (EBA), according to a study that also illustrates the variable clinical and immunological presentations ... Read more

November 5, 2018November 5, 2018

RegeneRX, GtreeBNT Provide Update on RGN-137 for Treatment of Epidermolysis Bullosa

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RegeneRx Biopharmaceuticals announced a clinical trial update on RGN-137, an investigational therapy for the treatment of epidermolysis bullosa (EB). The update was provided by GtreeBNT, RegeneRx’s partner for the ... Read more

October 31, 2018October 31, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand ... Read more

October 29, 2018October 29, 2018

FDA Advises Fibrocell on Phase 3 Trial Design for FCX-007, Potential RDEB Therapy

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Fibrocell Science has completed a type C meeting with the U.S. Food and Drug Administration in which they discussed the design of a Phase 3 clinical trial to assess the ... Read more

October 24, 2018October 24, 2018

TWi Biotechnology Enrolls First Patient in Phase 2 Trial Testing AC-203 for EB Treatment

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The first participant has been enrolled in a Phase 2 clinical trial testing AC-203 for the treatment of inherited epidermolysis bullosa (EB), the trial’s sponsor, TWi Biotechnology, announced. AC-203 (also ... Read more

October 22, 2018October 22, 2018

DNA Sequencing Technique Identifies Two New Mutations in Patient with Simplex and Junctional EB

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A technique used to decipher the DNA sequence, called next-generation sequencing (NGS), was successfully used to identify two new mutations in a patient diagnosed with both simplex and junctional ... Read more

October 17, 2018October 17, 2018

First RDEB Adult Patients Do Well in Clinical Trial with Gene Therapy KB103

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The first two adults with recessive dystrophic epidermolysis bullosa (RDEB) treated with the topical gene therapy candidate KB103 experienced increased levels of functional human collagen 7 (COL7) protein and anchoring ... Read more

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