Abeona Therapeutics Appoints Carsten Thiel, Ph.D., as Chief Executive Officer

Company strengthens management team with biopharmaceutical veteran bringing extensive commercial experience in life-threatening rare diseases Tim Miller, Ph.D. continues as president and assumes chief scientific officer role NEW YORK and CLEVELAND, A... Biopharmaceuticals, Personnel Abeona Therapeutics, gene therapy, epidermolysis bullosa, Sanfilippo syndrome
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news

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(Universidad Carlos III de Madrid) A group of researchers from the Biomedical Research Networking Centre on Rare Diseases (CIBERER), Universidad Carlos III de Madrid (UC3M), the Research Center for Energy, Environmental and Technology (CIEMAT), and the Instituto de Investigaci ó n Sanitaria Fundaci ó n Jim é nez D í az (IIS-FJD) have led a study which demonstrates the viability of a gene editing strategy for recessive dystrophic epidermolysis bullosa (also known as butterfly chilidren) with the tool CRISPR/Cas9 in preclinical models with this disease.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Planta Med DOI: 10.1055/a-0850-0224With central European approval in January 2016 for a betulin-oleogel (Episalvan), used to accelerate wound closure in partial thickness wounds, the herbal active ingredient triterpene dry extract (betulin), from birch bark, was introduced into therapy for the first time. Clinical evidence of accelerated wound healing was provided in a new study design by means of intraindividual comparison of split-thickness skin graft donor wounds and burn wounds. Clinical results of a phase II study evidencing accelerated wound healing in the rare disease epidermolysis bullosa are also available, and a ...
Source: Planta Medica - Category: Drugs & Pharmacology Authors: Tags: Reviews Source Type: research
(Universidad Carlos III de Madrid) A group of researchers lead by a lecturer from the Universidad Carlos III de Madrid (UC3M), Marcela del R í o, from the CIEMAT, the Rare Diseases Networking Biomedical Research Centre (Initials in Spanish: CIBERER-- ISCIII) and Fundaci ó n Jim é nez D í az has identified a common genetic signature among three rare skin diseases or genodermatoses: recessive dystrophic epidermolysis bullosa, Kindler syndrome and xeroderma pigmentosum. In the near future, these findings will allow efficient and safe evidence-based therapeutic approaches.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
Inherited epidermolysis bullosa (EB) is a clinically and genetically heterogeneous group of rare diseases characterized by skin and mucous membrane fragility. EB primarily involves the skin and, in specific subtypes, the mucous membrane, resulting in complications which can strongly affect nutritional status (e.g. gastrointestinal complications, hand deformities, pain). The aims of nutritional support mainly include improving nutritional status, alleviating the stress of oral feeding and minimizing nutritional deficiencies, thus consequently improving growth, pubertal development, bowel function, immune status and wound healing.
Source: Clinical Nutrition - Category: Nutrition Authors: Tags: Review Source Type: research
Rare diseases affect approximately 30 million people in the European Union and present a major health issue. Over 1000 rare skin diseases are known, many of which are of genetic origin and manifest in childhoo...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes aft...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Letter to the Editor Source Type: research
Epidermolysis bullosa acquisita (EBA) is an orphan autoimmune disease. Several clinical phenotypes have been described, but subepidermal blistering is characteristic of all variants. Limited data on clinical a...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Epidermolysis bullosa (EB) and autoimmune blistering diseases (AIBD) describe a group of rare chronic dermatoses characterized by cutaneous fragility and blistering. Although uncommon, significant ocular surfa...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Review Source Type: research
An inherited disease called epidermolysis bullosa destroyed the 7-year-old's skin. Scientists grew new skin in the lab without the genetic flaw, and replaced 80 percent of his skin. He is thriving.(Image credit: CMR Unimore/Nature )
Source: NPR Health and Science - Category: Consumer Health News Authors: Source Type: news
Conclusion This tool offers new perspectives in a translational context to identify patients for genetic research. Moreover, when new molecular bases are discovered, our strategy will help to identify additional eligible patients for genetic screening. Graphical abstract
Source: Journal of Biomedical Informatics - Category: Information Technology Source Type: research
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