Recent progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia.

Recent progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia. Curr Drug Targets. 2019 Jul 26;: Authors: Hamed EM, Meabed MH, Aly UF, Hussein RRS Abstract Thalassemia syndromes are a group of inherited blood disorders caused by limitation or absence of alpha or beta- globin chain synthesis resulting in ineffective erythropoiesis and chronic hemolytic anemia. According to the clinical fact of thalassemia as recessive autosomal, thalassemia divided into alpha and beta thalassemia. The main complication of beta thalassemia is excessive red blood cells destruction, oxidative stress, extramedullary hematopoiesis pseudotumors, hemosiderosis-induced organ dysfunction, malignancy, polyneuropathy, myopathy, osteoporosis, and leg ulcers. Although, the first obligatory therapy for thalassemia major is a blood transfusion, cause cardiac and hepatic hemosiderosis that is the most common cause of death in these patients. The targeted sequencing protocols include Hb F inducers, ineffective erythropoiesis correctors, antioxidant, vitamins, and herbals. Resveratrol is a novel herbal therapeutic approach for β-thalassemia. New appearance of molecular therapy in response to adverse effects of drugs. ActRIIB/IgG1 Fc recombinant protein is a novel molecular therapy in thalassemia. This review focuses light on maximizing the efficacy of unique combinations and new future therapeutic interventions in thalassemia that reverse splenomegaly, ...
Source: Current Drug Targets - Category: Drugs & Pharmacology Authors: Tags: Curr Drug Targets Source Type: research

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ConclusionsCompared to other pituitary adenomas, secreting and non-secreting, where pituitary surgery is the recommended first-line treatment, men with prolactinomas will usually respond to medical treatment with no need for any additional treatment.
Source: Pituitary - Category: Endocrinology Source Type: research
Conclusion: These data confirm the highly prevalence of comorbidities in COPD patients and their impact on pulmonary disability and prognosis.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Monitoring airway disease Source Type: research
This study will report the results of a series of descriptive and survival analyses compare an exposure of COPD in people over 40 years old (N=22,942) to predict the outcomes of: heart failure, depression, anxiety, coronary artery disease, diabetes, anemia, hypertension, ischemic heart disease, underweight, and osteoporosis while controlling for EHR status of: smoking, age, sex, and rurality. Results will be computed in March 2019. By the date of the ERS Congress 2019, we will be able to report the results of the aforementioned analyses, which will demonstrate the utility of using large EMR datasets to illustrate comorbid ...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: General practice and primary care Source Type: research
AbstractThe termLate ‐onset hypogonadism (LOH) was coined in 2002 and defined as a disease entity in the ISA, ISSAM, EAU, EAA and ASA endorsed Recommendations for Investigation, Treatment and Monitoring of LOH (2005 and 2008) as ‘a clinicaland biochemical syndrome associated with advancing age, characterized by symptomsand a deficiency in serum testosterone (T) ’. LOH was classified as a combined primary and secondary hypogonadism since the endocrine capacity of the testes and the pituitary are impaired. Symptoms of LOH include loss of libido, erectile dysfunction, loss of muscle mass, increased body fat, ane...
Source: Andrology - Category: Urology & Nephrology Authors: Tags: Review Article Source Type: research
AbstractPurpose of ReviewThe significance and roles of marrow adipose tissue (MAT) are increasingly known, and it is no more considered a passive fat storage but a tissue with significant paracrine and endocrine activities that can cause lipotoxicity and inflammation.Recent FindingsChanges in the MAT volume and fatty acid composition appear to drive bone and hematopoietic marrow deterioration, and studying it may open new horizons to predict bone fragility and anemia development. MAT has the potential to negatively impact bone volume and strength through several mechanisms that are partially described by  inflammaging...
Source: Current Osteoporosis Reports - Category: Orthopaedics Source Type: research
AbstractThe termLate ‐onset hypogonadism (LOH) was coined in 2002 and defined as a disease entity in the ISA, ISSAM, EAU, EAA and ASA endorsed Recommendations for Investigation, Treatment and Monitoring of LOH (2005 and 2008) as “a clinicaland biochemical syndrome associated with advancing age, characterized by symptomsand a deficiency in serum testosterone (T) ”. LOH was classified as a combined primary and secondary hypogonadism since the endocrine capacity of the testes as well as the pituitary are impaired. Symptoms of LOH include loss of libido, erectile dysfunction, loss of muscle mass, increased body f...
Source: Andrology - Category: Urology & Nephrology Authors: Tags: REVIEW ARTICLE Source Type: research
Conclusions: Based on this study, males and females with ACTH-dependent Cushing’s syndrome present different clinical patterns. However, these differences do not justify different diagnostic strategies or treatment based on sex, considering the similar surgical outcome. Clinicians should be alert to diagnose accompanying osteoporosis (with fractures) in male patients with ACTH-dependent Cushing’s syndrome.
Source: Frontiers in Endocrinology - Category: Endocrinology Source Type: research
Authors: Bilezikian JP, Hattersley G, Mitlak BH, Hu MY, Fitzpatrick LA, Dabrowski C, Miller PD, Papapoulos SE Abstract Objective: To evaluate, post hoc, the efficacy and safety of abaloparatide by degree of renal impairment. Methods: ACTIVE was a phase 3, 18-month, randomized, double-blind, active-comparator, placebo-controlled study of postmenopausal women with osteoporosis who received subcutaneous abaloparatide 80 µg, placebo, or open-label teriparatide 20 µg daily. Patients with serum creatinine>2.0 mg/dL or 1.5-2.0 mg/dL with an estimated glomerular filtration rate (eGFR)
Source: Current Medical Research and Opinion - Category: Research Tags: Curr Med Res Opin Source Type: research
Abstract Purpose: The purpose of this retrospective study was to investigate the relationships of types of dental insurance coverage in Korea with sociodemographic characteristics and the prevalence of systemic and oral diseases, as well as to evaluate the socioeconomic impact of Korean dental insurance policies. Methods: Sample cohort data from 2006 to 2015 were obtained from the National Health Insurance Service. Patients were divided into 2 groups. The exposed group comprised patients who received insurance benefits for complete dentures, removable partial dentures, and implant care, while the control grou...
Source: Journal of Periodontal and Implant Science - Category: Dentistry Authors: Tags: J Periodontal Implant Sci Source Type: research
Publication date: Available online 30 July 2019Source: Revista Clínica Española (English Edition)Author(s): M. Rincón Gómez, C. Hernández Quiles, M. García Gutiérrez, J. Galindo Ocaña, R. Parra Alcaraz, V. Alfaro Lara, R. González León, M. Bernabeu Wittel, M. Ollero BaturoneAbstractIntroductionHip fracture in the elderly is one of the most prevalent diagnoses in Orthopedic Surgery Departments. It has a great impact in medical, economic and social terms. Our objective is to analyze clinical impact of a co-management care model between orthopedic surgery a...
Source: Revista Clinica Espanola - Category: Internal Medicine Source Type: research
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