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Fabry Disease (FD) is a rare X-linked lysosomal storage disorder (LSD) caused by mutations in the GLA gene coding for the enzyme alpha-galactosidase A (α-GAL A). As a consequence globotriaosylceramide (Gb3), the enzyme's substrate, is not metabolized efficiently. The result is progressive
This is a pilot study with a descriptive study design.
Anthracycline induced late onset cardiotoxicity, defined in terms of abnormal findings on echocardiography, has been reported to occur in 57% of childhood cancer survivors. Serial monitoring of cardiac function by means of echocardiography
Part A: Patients who received a high dose of anthracyclines and who completed chemotherapy for a minimum of 2 year and are still in remission we gathered information about their previous heart disease, radiation dose to the heart, bone marrow transplant, age at treatment, gender, elevation of serum
Materials and Methods: will be conducted an interventional, randomized, double-blind, placebo-controlled study. The series will consist of patients with at least 18 years old, suffering from chronic kidney disease stage V on dialysis and mean residential blood pressure superior to 135 x 85 mm Hg
Patients who successfully complete Fx1B-201 will report to the clinical unit on Day 0 to sign the informed consent form and determine eligibility for Protocol Fx1B-303. In addition, on Day 0, patients will have their entrance criteria reviewed, and medical histories and demographic characteristics
Cardiovascular diseases (CVD) are the main causes of death and hospitalization in patients affected by ESRD . The risk of death from CVD is already detectable in the early steps of chronic renal failure and it is from 20 to 30 times higher than in the general population. In ESRD patients the
Inclusion Criteria:
1. Age≥18 years and less than 75 years
2. Essential hypertension patients
3. Sedentary systolic blood pressure≥140mmHg and less than 180mmHg, and/or sedentary diastolic blood pressure≥90mmHg and less than 110mmHg
4. Reproductive women agree to take a reliable contraception
II. OBJECTIVES
II.A. Primary Objectives: To evaluate response rate (RR) and overall survival (OS).
II.B. Secondary Objective: To evaluate the time to progression (TTP), median time to response (MTR), toxicity and quality of life (QOL).
III. STUDY DESIGN:
III.A. Inclusion criteria:
1. Written
INTRODUCTION
BACKGROUND: In recent years, the need to achieve increasingly ambitious therapeutic goals for dyslipidemias has prompted the search for more potent pharmacological agents to lower circulating atherogenic lipoprotein concentrations and enhance reverse cholesterol transport (RCT). While