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Efficacy and Safety of Gene Therapy Using Adeno-Associated Virus Vector in Patients with Choroideremia
Choroideremia (CHM) is a rare genetically conditioned retinal disease caused by mutations in the CHM gene, leading to a lack of production of the protein REP1 (ras-associated binding escort protein 1). Currently, there is no approved treatment for CHM. The aim of the study (Tübingen Choroideremia Gene Therapy) published last year in the journal JAMA Ophthalmology was to evaluate the safety and efficacy of subretinally applied gene therapy using an adeno-associated virus vector (AAV2), designed to deliver a functional copy of the CHM gene to…
Results of ATTUNE Total Knee Replacement Implantation – Data from the National Joint Registry for England, Wales, Northern Ireland and the Isle of Man
The ATTUNE knee replacement system was fully introduced into clinical practice in 2013. Since then,…
Young man with nephrotic syndrome and portal vein thrombus successfully treated with edoxaban − case report
Venous thrombosis is one of the main complications of nephrotic syndrome (NS), though it is most…
Options of Anticoagulant Therapy During Pregnancy: Focus on Fondaparinux
The use of most medicinal products is limited during pregnancy either by known teratogenic effects…
Alternative to benzodiazepines or clomethiazole in the treatment of alcohol withdrawal syndrome?
Alcohol withdrawal syndrome (AWS) is a potentially life-threatening condition. A meta-analysis…
Importance of pH in Care for Stoma Patients
Maintaining good quality peristomal skin and preventing complications related to the stoma site are…
Combination of SGLT2 inhibitors with metformin in terms of mechanism of action
In the treatment of type 2 diabetes (T2D), metformin is recommended as the first-line drug alongside...
Immunotolerance is still the goal of management of hemophilia A with inhibitor in the era of non-factor therapy
New molecules in hemophilia therapy have raised many questions, including regarding the…
Basic Information on the Spread and Course of COVID-19 Infection in China: What We Know So Far?
Currently, we are experiencing a worldwide outbreak of COVID-19 infection caused by the SARS-CoV-2…
Rapid Diagnosis of Depression in Primary Care Settings
Depression is one of the most commonly occurring psychiatric disorders encountered in general…
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