Exton firm advancing drug candidate to help 'butterfly children'

Fibrocell Science Inc., a Chester County gene and cell therapy company, is making progress on its experimental treatment to help “butterfly children.” The Exton company’s lead drug candidate, FCX-007, is being developed to treat a condition known as recessive dystrophic epidermolysis bullosa (RDEB). The rare skin disease afflicts an estimated 1,100 to 2,500 people, mostly children, nationwide. The condition prevents t he layers of skin from forming properly. As a result, pediatric patients…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news

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Abstract Epidermolysis bullosa (EB) is a group of rare inherited bullous skin disorders that differ in nature and severity. Currently, there is no cure for the disease. One of the complex problems of EB is the repetitive and painful care of skin wounds. The purpose of this study was to explore how adult patients and parents experienced the impact of wound care during childhood and which coping strategies they considered as helping. A qualitative study was performed, comprising semi-structured in-depth interviews with 7 adult patients and 6 parents. The impact, physically, psychologically and on daily life, was app...
Source: Acta Dermato-Venereologica - Category: Dermatology Authors: Tags: Acta Derm Venereol Source Type: research
Authors: Hofmann SC, Weidinger A Abstract Epidermolysis bullosa acquisita (EBA) is a rare acquired subepidermal bullous autoimmune dermatosis, associated with autoantibodies against collagen type VII, the most important component of dermal anchoring fibrils. Blister induction occurs after binding of autoantibodies to collagen type VII, leading to complement activation, recruitment of neutrophils and secretion of proteases. Clinically, the disease is mostly characterized by tense blisters on trauma-exposed body areas which heal with scarring (mechanobullous form of EBA). The second most frequent subty...
Source: Der Hautarzt: Zeitschrift fur Dermatologie, Venerologie, und verwandte Gebiete - Category: Dermatology Tags: Hautarzt Source Type: research
We report the case of a girl with epidermolysis bullosa simplex (EBS) associated with muscular dystrophy secondary to congenital plectin deficiency. She presented severe respiratory tract lesions extending from the oral cavity to the larynx. In particular, we describe our medical and surgical management of the laryngeal lesions, responsible for several episodes of respiratory distress and feeding difficulties.DiscussionEpidermolysis bullosa simplex associated with muscular dystrophy is a rare hereditary form of EB, as fewer than 50 cases have been reported in the literature. This form is characterized by mucosal lesions in...
Source: European Annals of Otorhinolaryngology, Head and Neck Diseases - Category: ENT & OMF Source Type: research
ConclusionscSCC is a life-threatening complication of RDEB patients. Although tumors are usually well differentiated, they tend to relapse. This is the first Spanish report of cSCC arising in RDEB patients.
Source: Clinical and Translational Oncology - Category: Cancer & Oncology Source Type: research
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
(Thomas Jefferson University) New research lays foundation for upcoming clinical trial for patients with epidermolysis bullosa.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
CONCLUSIONS: These data support a "first in RDEB" phase II clinical trial of rigosertib to assess tumor targeting in patients with late stage, metastatic and/ or unresectable SCC. PMID: 30846478 [PubMed - as supplied by publisher]
Source: Clinical Cancer Research - Category: Cancer & Oncology Authors: Tags: Clin Cancer Res Source Type: research
CONCLUSIONS: PTCy BMT in RDEB provides a means of attaining immunotolerance for future donor-derived cellular grafts (trial registered as NCT02582775). This article is protected by copyright. All rights reserved. PMID: 30843184 [PubMed - as supplied by publisher]
Source: The British Journal of Dermatology - Category: Dermatology Authors: Tags: Br J Dermatol 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
CONCLUSIONS: Guidelines regarding implant treatment of patients with oLp, Pe, EB, SjS, sLE or sSc do not exist nor are contraindicating conditions defined. Implant survival rates of patients affected are comparable to those of healthy patients. For implant-prosthetic rehabilitation of patients with Pe and sLE no conclusions can be drawn due to lack of sufficient clinical data. Implant-prosthetic treatment guidelines regarding healthy patients should be strictly followed, but frequent recall is recommended in patients affected with oLp, SjS, EB, SSc, Pe or sLE. PMID: 30818315 [PubMed - in process]
Source: Medicina Oral, Patologia Oral y Cirugia Bucal - Category: ENT & OMF Tags: Med Oral Patol Oral Cir Bucal Source Type: research
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