Therapy of Genetic Disorders: Novel Therapies for Duchenne Muscular Dystrophy
Abstract
Duchenne muscular dystrophy is an inherited, progressive muscle-wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin production and to enhance or preserve muscle mass. Gene therapy methods are being developed to replace the defective dystrophin gene or induce dystrophin production from mutant genes. Stem cell approaches are being developed to replace lost muscle cells while also bringing in new dystrophin genes. This review summarizes recent progress in the field with an emphasis o...
Source: Current Pediatrics Reports - June 1, 2014 Category: Pediatrics Source Type: research
Future Therapies for IgE-Mediated Food Allergy
Abstract
Food allergy is prevalent, affecting approximately 4–8 % of children. There is no currently approved treatment for food allergy, and while strict allergen avoidance is recommended it is difficult to achieve. Therefore, accidental exposures and reactions are common. There is an urgent need for the development of therapeutic approaches that will improve the health and quality of life of children with food allergy. The majority of current clinical research focus is on specific food allergen immunotherapy through oral, sublingual, or epicutaneous routes. Pre-clinical research has focused on maki...
Source: Current Pediatrics Reports - June 1, 2014 Category: Pediatrics Source Type: research
Non-IgE-Mediated Food Allergy: FPIES
Abstract
Food protein-induced enterocolitis syndrome (FPIES) is a non-IgE-mediated food hypersensitivity with usual onset in infancy. The most common FPIES triggers are cow milk, soy and rice; in addition, oats, vegetables, egg, poultry and seafood have been reported. In the acute form, when food is ingested on an intermittent basis or following a period of avoidance, FPIES presents with profuse vomiting within 1–3 h after ingestion, occasionally accompanied by diarrhea and dehydration. In the chronic form, when food is ingested on a regular basis, FPIES presents with intermittent vomiting, diarrhea,...
Source: Current Pediatrics Reports - June 1, 2014 Category: Pediatrics Source Type: research
IgE-Mediated Food Allergy: Current Management
Abstract
Food allergy affects 4–8 % of children, and it appears to be increasingly common. The prevalence of peanut allergy has increased in the US and internationally. Pediatricians are on the front lines of providing patient care, so disseminating guidelines and current information regarding appropriate evaluation and principles of management to them is critically important. The approach to the patient with suspected food allergy requires a detailed medical history. In vitro testing is widely available but should be ordered judiciously. Using the history to guide testing can help avoid potential pi...
Source: Current Pediatrics Reports - June 1, 2014 Category: Pediatrics Source Type: research
Non-IgE Medicated Food Allergy: Eosinophilic Esophagitis Update on the Pathogenesis, Clinical Features, and Management of Eosinophilic Esophagitis in Children
Abstract
Eosinophilic esophagitis (EoE) is a chronic allergic disorder of increasing worldwide prevalence. Diagnosis and management require upper gastrointestinal endoscopy with biopsy. Therapeutic strategies in children and adults include empiric and testing-based elimination diets as well as topical esophageal corticosteroids. The last 12 months have provided a number of insights including updated diagnostic/management guidelines, the role of PPI therapy, and an understanding that EoE disease mechanisms rely on basophils and mast cells in addition to eosinophils. Novel diagnostic techniques have been...
Source: Current Pediatrics Reports - June 1, 2014 Category: Pediatrics Source Type: research
