This trial is evaluating whether Treatment will improve 1 primary outcome and 1 secondary outcome in patients with Fabry Disease. Measurement will happen over the course of 3 years.
This trial requires 250 total participants across 0 different treatment group
"This cohort study supports the hypothesis that Fabry disease is caused by a defect of the enzyme alpha-galactosidase A. There is, however, evidence that only a minority of patients with alpha-galactosidase A deficiency have clinical manifestations of Fabry disease." - Anonymous Online Contributor
"For the first time, a cure for Fabry disease was determined. Clinical and biochemical parameters remained stable 6 months after the cessation of the beta-glucosidase inhibitor agalsidase beta. The reduction in enzyme activities and the normalization of Fabry disease biomarkers in the circulation indicate that agalsidase beta treatment can indeed lead to improvement of the disease. Further studies are necessary to confirm our findings." - Anonymous Online Contributor
"With the exception of one population, there were no significant differences for the prevalence of FD in various age ranges. There was one case of FD in the US per 250 000 inhabitants per year. Therefore, the prevalence of FD is 1.1 per million inhabitants per year. The prevalence of FD is about 1.6 cases per million inhabitants at any given time. The prevalence of FD shows considerable variability in different geographic regions, with highest prevalence in the Middle East. There was no difference among those with and without diabetes." - Anonymous Online Contributor
"Although Fabry disease can present with any systemic disease, men with Fabry disease more often present with systemic symptoms such as myalgia, and women with Fabry disease present more often with a family history of heart disease. Fabry disease patients are less likely to have angiographic evidence of atherosclerosis, and they have a longer time of course and a lower frequency of cardiac events than systemic vasculitis. In symptomatic patients, Fabry disease can also present with angiographically demonstrable small vessel involvement. Patients with Fabry disease often present with gastrointestinal symptoms such as angina or diarrhea. These patients more often present with severe anemia, peripheral neuropathy, and renal disease compared to patients without Fabry disease." - Anonymous Online Contributor
"There are no approved treatments for fabry disease. Because of the debilitating nature of the disease, supportive, personalized patient-centered care often is necessary. Because of the high risk of sudden death from cardiac complications due to cardiovascular disease in this group, periodic cardiac evaluations and monitoring is highly recommended. Long-term management includes monitoring of cardiovascular risk factors and the use of aspirin for people with acute coronary syndrome who are at higher than usual risk." - Anonymous Online Contributor
"Fabry disease is a kidney or heart disease that is due to inherited abnormalities in the enzyme aldolase B. It is an X-linked recessive disease that is associated with sensorineural hearing loss.\n" - Anonymous Online Contributor
"[A combination of ERT and IFN-α2a treatment is sufficient to stop renal damage and to prevent cardiovascular events] (https://www.bioglan.com/pages/fabry-treatment). If you are a patient with Fabry disease or familial Fabry disease"
"Chiton (disambiguation)\n\nThe name Chiton is used in different ways; the most common is to describe specific species of gastropod." - Anonymous Online Contributor
"The latest published papers that relate to Fabry disease will be published in medical journals, research papers, and in the ‘Cancer Research UK’ magazine. It is important to keep receiving these recent updates as [research can help make sure Fabry disease remains the most comprehensively searched, best-understood and treated form of the hereditary [protein] disorders]. To receive these updates visit Power (http://www.withpower.com/clinic_trials)." - Anonymous Online Contributor
"Most of the patients were treated using a combination of treatment. In patients treated with corticosteroid alone, a low percentage was managed to respond satisfactorily. The effectiveness of combined treatment seemed to be similar and acceptable for patients with or without renal involvement." - Anonymous Online Contributor
"In patients with Fabry disease, renal disease is present at an earlier age than the general population. The mean age of disease manifestation is 17.2 yr. Fabry disease is diagnosed in both males and females." - Anonymous Online Contributor
"There were no obvious correlations between the side effects and the diseases being treated with the medications. Most side effects were mild and tolerable, with a small percentage only affecting the specific individuals in the cases of allergic reaction, hypersensitivity, or other special side effects. Many other side effects may not be reported on medical records, as the side effects often only pose minor negative impact on patients." - Anonymous Online Contributor
"Fabry disease patients that received and who had been on treatment before enrollment showed significantly greater decreases in MDA and improved GFR, CKD, and EF by 2 years of treatment, but no improvements in ECG in patients receiving a placebo." - Anonymous Online Contributor