Epidermolysis Bullosa News and Research

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Researchers reveal how butterfly disease patients develop cutaneous squamous cell carcinomas

Fragile skin that blisters easily: 90 percent of the patients that suffer from the skin condition recessive dystrophic epidermolysis bullosa (RDEB) develop rapidly progressing cutaneous squamous cell carcinomas, a type of skin cancer, by the age of 55. 80 percent of these patients will die due to metastasis within five years after the cancer has been first detected.

11 Mar 2016

Positive interim results from TWIB's AC-201 CR Phase 2 study for hyperuricemia and gout

TWi Biotechnology Inc. today announced interim results from the ongoing Phase 2 proof of concept clinical study evaluating AC-201 CR as an oral uricosuric and anti-inflammatory agent for the treatment of hyperuricemia and prevention of gout flares combining with febuxostat, a xanthine oxidase inhibitor.

24 Dec 2015

FDA awards research grants to boost product development for patients with rare diseases

The U.S. Food and Drug Administration today announced it has awarded 18 new research grants totaling more than $19 million to boost the development of products for patients with rare diseases, which affect the lives of nearly 30 million Americans.

11 Oct 2015

New stem-cell based therapy provides pain relief, reduces severity of RDEB in children

Promising results from a trial of a new stem-cell based therapy for a rare and debilitating skin condition have been published in the Journal of Investigative Dermatology. The therapy, involving infusions of stem cells, was found to provide pain relief and to reduce the severity of this skin condition for which no cure currently exists.

27 May 2015

TWi Biotechnology obtains Rare Disease Drug designation in Taiwan for use of AC-203 to treat EBS

TWi Pharmaceuticals, Inc. today announced that its fully owned subsidiary, TWi Biotechnology, Inc., has received the designation of Rare Disease Drug by Taiwan FDA for use of AC-203 to treat Epidermolysis Bullosa Simplex (EBS), and is eligible for applying for coverage under National Health Insurance Administration.

10 Mar 2015

New strategy may ensure safety of adult epidermal stem cells before performing treatments

A team of European researchers has devised a strategy to ensure that adult epidermal stem cells are safe before they are used as treatments for patients. The approach involves a clonal strategy where stem cells are collected and cultivated, genetically modified and single cells isolated before being rigorously tested to make sure they meet the highest possible safety criteria.

28 Feb 2015

Stem cell-based gene therapy shows long-term outcomes for treatment of genetic skin diseases

Stem cell-based gene therapy holds promise for the treatment of devastating genetic skin diseases, but the long-term clinical outcomes of this approach have been unclear.

27 Dec 2013

Oxford Gene Technology releases next generation sequencing survey results

Oxford Gene Technology, provider of innovative genetics research and biomarker solutions to advance molecular medicine, has released the results from a recent survey of researchers using next generation sequencing.

From Oxford Gene Technology 21 Aug 2013

Walmart stores join together to help Cincinnati Children's battle epidermolysis bullosa disease

This year, 32 Tri-State Walmart stores are joining together to help Cincinnati Children's battle a rare, devastating disease: Epidermolysis Bullosa (EB).

21 Aug 2013

Researchers find new way to repair genetic defects in the skin cells of epidermolysis bullosa patients

A research team led by pediatric blood and marrow transplantation experts Mark Osborn, Ph.D. and Jakub Tolar, M.D., Ph.D. from the Masonic Cancer Center, University of Minnesota, have discovered a remarkable new way to repair genetic defects in the skin cells of patients with the skin disease epidermolysis bullosa.

10 Jun 2013

Scioderm gets Breakthrough Therapy designation for SD-101 from FDA for treatment of EB

Scioderm announced its investigational product SD-101 has received Breakthrough Therapy designation by the U.S. Food and Drug Administration for the treatment of patients with inherited Epidermolysis Bullosa.

5 May 2013

FDA allows Scioderm’s SD-101 IND application for Epidermolysis Bullosa to proceed

Scioderm announced that the US Food and Drug Administration (FDA) has reviewed and allowed the investigational new drug (IND) application for SD-101 to proceed. SD-101 is a topical treatment being developed for the treatment of Epidermolysis Bullosa (EB).

14 Feb 2013

Shire commences ABH001 Phase 3 study in Epidermolysis Bullosa

Shire plc, today announced the initiation of a Phase 3 study designed to evaluate the efficacy and safety of ABH001, its dermal substitute therapy, for the treatment of non-healing wounds in patients with Epidermolysis Bullosa (EB), a group of rare genetic skin disorders that begin to manifest at birth or early childhood and occur in approximately 19 per 1 million live births in the US.

10 Feb 2013

Researchers to develop new diagnostic tools and treatments for people with rare diseases

A multi-million Euro initiative is bringing together researchers from across the world to develop new diagnostic tools and new treatments for people with rare diseases and to connect research data in this area on a global scale.

25 Jan 2013

Tβ4 offers both neurorestoration and neuroprotection after TBI

RegeneRx Biopharmaceuticals, Inc. reported that data showing that Thymosin beta 4 (Tβ4) "provides both neuroprotection and neurorestoration after traumatic brain injury (TBI)," was published in the current (May 2012) edition of the Journal of Neurosurgery, 116:1081-1092.

28 May 2012

RegeneRx wins four patents in Israel, Mexico

RegeneRx Biopharmaceuticals, Inc. announced today that the Company has received an issued patent in Mexico and allowances of three additional patent applications in Mexico and Israel.

10 Apr 2012

RegeneRx's RGN-259 safe, well tolerated in patients with dry eye syndrome

RegeneRx Biopharmaceuticals, Inc. announced today that it has received the clinical study report for its recently completed Phase 2 clinical trial evaluating RGN-259 for the treatment of dry eye syndrome.

5 Jan 2012

Towards a national plan of action for rare diseases

"Rare diseases", by their very definition, occur in no more than 5 people out of every 10,000 inhabitants. Barely noticed by the general public, only around 1,000 of the currently 6,000 or so different rare diseases currently listed on the Internet platform Orphanet are treatable nowadays.

5 Dec 2011

Two RegeneRx cardiac-related patents receive notice of allowance in Australia, Mexico

RegeneRx Biopharmaceuticals, Inc. announced today that the Company has been notified that two cardiac-related patents will be allowed in Australia and Mexico.

18 Nov 2011

Positive data from RegeneRx RGN-259 Phase 2 trial on dry eye syndrome

RegeneRx Biopharmaceuticals, Inc. today announced positive data from its 72-patient, Phase 2 clinical trial with RGN-259, a sterile, preservative-free eye drop, to evaluate its safety and efficacy for the treatment of signs and symptoms of dry eye syndrome over a treatment period of 30 days using the Controlled Adverse Environment (CAE) model.

5 Nov 2011