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The main purpose of this study was to determine the safety and tolerability of 3 different doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also evaluated the effects of duvoglustat on functional parameters in Pompe disease.

Background: * Carcinoembryonic antigen (CEA) is a protein present mostly in cancer cells. * An experimental procedure developed for treating patients with cancer uses blood cells found in their tumors or bloodstream. These cells are genetically modified using the anti-CEA gene a…

This study will collect information about markers of inflammation, blood clotting and blood vessel function in HIV-infected adults and healthy volunteers. Biomarkers are biological indicators that have been associated with disease. Certain markers of inflammation, blood clotting,…

This study will determine if the level of virus in HIV-infected patients taking antiretroviral medications for prolonged periods decreases or persists at a stable level. It will also examine whether new gene changes (mutations) occur during drug suppression. HIV-infected patient…

Cirrhosis is characterized by loss of muscle as well as fat mass, which increases morbidity and mortality before, during, and after liver transplantation. A common mechanism for the reduced muscle and fat mass in cirrhosis is an increased expression of the TGF (transforming growt…

FemmeJock is a pelvic floor support system developed by pelvic floor physiotherapists for patients with pelvic organ prolapse in order to ameliorate symptoms of pelvic floor pressure and discomfort. The device is a girdle to be worn on the outside of underwear. The device is mach…

Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to stimulate weight and muscle growth which will maximize nutritional potential and improve overall quality of life. We theorize that this will occur through a multitude of factors: increased ap…

The National Pregnancy Registry for Psychiatric Medications is dedicated to evaluating the safety of psychiatric medications such as antidepressants, ADHD medications, sedative hypnotics, and atypical antipsychotics that many people take during pregnancy to treat a wide range of …

TOTEME offers to treat certain chronic strictures of the ureter with MEMOKATH 051 to assess its effectiveness, tolerance and mean durability during a 3 years follow up period. Patients usually treated with double J stents will be included. The double J stent will be replaced by …

Pheochromocytoma or paraganglioma are tumors generating hypertension as a symptom. Different biological tests are currently available to diagnose these tumors. However, they all lack specificity since they do not distinguish cases of hypertension without pheochromocytoma or parag…

ICU-acquired weakness represents a common and often devastating disease process which affects greater than 50% of critically ill patients. This pathogenesis of this acquired disease is multifactorial and results in variable severity, ranging from mild, transient to severe, perman…

This study will test the reliability of a procedure for self-administering ethanol (alcohol) intravenously (through a vein), using a computer-assisted method. People ordinarily self-administer alcohol through drinking alcoholic beverages, but blood alcohol levels resulting from d…

The purpose of this phase I randomised cross over study is to determine and compare the bioavailability of two different oral formulations of AZD2281 in advanced solid tumour cancer patients

The purpose of this study is to evaluate safety/tolerability and clinical utility of low-dose transdermal therapeutic system (TTS \[medicated adhesive pad that is placed on the skin to deliver a timed-release dose of medication through the skin into the bloodstream\]) - fentanyl …

Latinos with diabetes who are attending the Emory Latino Diabetes education program will be approached about consenting to have clinical measurements collected to assess changes between baseline and monthly follow up sessions. The no-cost educational sessions follow the self-care…

There is currently no effective treatment for late infantile MLD once clinical symptoms are evident. METAZYM is a recombinant human arylsulfatase A developed for an intravenous ERT for the treatment of late infantile MLD. The overall objective of this study is to evaluate the eff…

The clinical, radiographic, and HRQL outcomes will be compared in operative and nonoperative adult spinal deformity patients.

To evaluate effectiveness of a prefixed 50% N2O- 50%O2 mixture in legal abortion under paracervical block.

The investigators observed that Propranolol, a beta-blocker commonly used in children was efficient to control the growth of alarming hemangiomas of the face. The primary objective of this study is to determine the efficiency of 1 month-early treatment of propranolol in infants …

Inflammatory or degenerative diseases of the brain and spinal cord, such as multiple sclerosis, may be related to problems with an individual s immune system. However, more information is needed on the ways in which the cells of the immune system interact with the central nervous…