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Clinical Trials About "Amicus Therapeutics Submit Japanese Drug Application Migalastat Fabry" RSS

13:34 EST 10th December 2018 | BioPortfolio

We list hundreds of Clinical Trials about "Amicus Therapeutics Submit Japanese Drug Application Migalastat Fabry" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

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Showing "Amicus Therapeutics Submit Japanese Drug Application Migalastat Fabry" Clinical Trials 1–25 of 7,100+

Extremely Relevant

MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study

This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.


Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

The purpose of this study is to compare the effect of AT1001 (migalastat hydrochloride) versus placebo on kidney GL-3.

German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.

The objective of the study is to document long term data on treatment with Migalastat under "real world" conditions. The selection of patients is based on the SmPC/Fachinformation. The study duration/patient will be 2 years.


A 12-Week Safety and Pharmacodynamic Study of AT1001 in Female Patients With Fabry Disease

The purpose of this study is to determine whether AT1001 (migalastat hydrochloride) is safe and effective in female patients with Fabry disease.

A Study of AT1001 in Patients With Fabry Disease

The purpose of this study is to determine whether AT1001 (migalastat hydrochloride) is safe and effective in patients with Fabry disease.

A 12-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease

The purpose of this study is to collect information on the safety of AT1001 (migalastat hydrochloride) and how AT1001 works in patients with Fabry disease.

A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease

The purpose of this study is to collect information on the safety of AT1001 (migalastat hydrochloride) and how AT1001 works in patients with Fabry disease.

Relevant

Fabry Outcome Survey (FOS)

The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease. The data from FOS may provide guidance to healthcare professionals about disease treatment options.

Fabry Disease Registry

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Registry are: - To enhance the understanding of the variability, progression, and ...

Evaluation of Phenotypic Variability in Fabry Disease

Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels. Understanding how the genotype influences the phenotype or clinical presentation can help us understand which patients are at risk for the complications of Fabry disease. This study aims to follow the natural history of this disease will help us understand and predict long-term outcomes for patients.

Diagnostic Value of the Electrocardiogram in Fabry Disease

Cardiac complications occur in 78% of patients with Fabry disease and are mainly characterized by a high frequency of left ventricular hypertrophy resulting from an accumulation of GL3 in cardiomyocytes. Apart from family screening, left ventricular hypertrophy is an important factor in the diagnosis of Fabry disease. This left ventricular hypertrophy is more often concentric and homogeneous, but it can also be asymmetric and mimic the patterns seen in so-called familial hypert...

Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation

New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease. In this case certain fat molecules are not digested and broken down by the body - but remain in the cells. These fat molecules build up to dangerous levels, which start to damage the body, because they accumulate e.g. in the walls of the blood vessels. This accumulation in the blood vessels of the whole body may c...

Relation Between Polymorphism in Genes Related to Kidney Disease and Renal Manifistations in Fabry Disease

The rational of the study is to try to predict which of the patients who suffer from Fabry disease will have End Stage Renal Disease. We hope to find correlation between certain modifier genes, previously related to renal disease, to renal disease in Fabry.

Biomarker for Patients With Fabry Disease

Development of a new MS-based biomarker for the early and sensitive diagnosis of a Fabry disease from plasma

Viennese Prevalence Study of Anderson-Fabry Disease

The prevalence of Anderson - Fabry disease in patients with left ventricular hypertrophy is unclear. The investigators will examine urine - α - Galactosidase activity and globotriaosylceramide isoforms in these patients.

Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease

Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Clearance of Gb3 and stabilization of renal function has been demonstrated in male patients treated with agalsidase beta (FABRAZYME). In contrast, no randomized, controlled study of the efficacy of recombinant al...

Extension Study of 1 mg/mL Pegunigalsidase Alfa in Patients With Fabry Disease

The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30.

Study to Learn How the Drug Aflibercept Works in in Japanese Patients With Increased Eye Pressure That is Caused by New Blood Vessels Growing in the Eye (Neovascular Glaucoma or NVG). Safety of the Drug and Patients' Tolerability of the Drug Injection is

The researchers in this trial want to learn how the drug aflibercept works in in Japanese patients with increased eye pressure that is caused by new blood vessels growing in the eye (neovascular glaucoma or NVG). They also want to find out how patient tolerate the application of the drug that is injected in the vitreous humor of the eye and if this will cause any medical problems during the trial (vitreous humor, also called vitreous body is the clear gel that fills the space b...

Fabry : National Initiative of Screening

Fabry disease is a genetic disease due to an enzymatic deficit. A screening of this disease allows patients to benefit from an enzyme replacement therapy and prevent the occurrence of life threatening manifestations such as an ischemic stroke. The purpose of the study is to determinate the prevalence of Fabry disease in a population of male patients hospitalized for an ischemic stroke. This study, with a screening of Fabry disease, allows the patients to make a precise ...

Lipidomics and Functional Analyses of Platelets in Fabry Disease

This study aims to evaluate whether platelets are biochemically and functionally altered in Fabry disease (FD) and therefore possibly implicated in FD manifestations such as cerebrovascular events. To test this hypothesis the investigators aim to compare platelet and plasma lipid profiles, as well as platelet function and coagulation parameters of FD patients and healthy controls.

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.

Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)

The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR.

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

The objective of PB-102-F51 is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Fabry Screening Study

To determine if patients with a deficiency of alpha-galactosidase A are at-risk for cardiac complications that commonly occur in the general population

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

Patients will undergo a SmartPill test to gain additional understanding of Fabry disease manifestation via motility abnormalities in order to improve symptom targeted therapy. An additional Endoscopic mucosal resection may be performed on further qualifying patients. Tissue analysis from this biopsy will include evaluation of abnormalities of cellular structure and morphology with correlation with gastrointestinal complaints for each patient and comparison against age matched n...


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