ReviewDrug design, development and therapy2011
Update on role of agalsidase alfa in management of Fabry disease.
Review in Drug design, development and therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- Pooled it
- Unexpected Hypotension in a Female Patient with Fabry Disease: Switching from Agalsidase α to β after Long-term ERT.Internal medicine (Tokyo, Japan) · 2025Article
- Article
- Case report: enzyme replacement therapy for Fabry disease presenting with proteinuria and ventricular septal thickening.BMC nephrology · 2024Article
- Cardiac Imaging in Anderson-Fabry Disease: Past, Present and Future.Journal of clinical medicine · 2021Review
- Auditing the frequency and the clinical and economic impact of testing for Fabry disease in patients under the age of 70 with a stroke admitted to Saint Vincent's University Hospital over a 6-month period.Irish journal of medical science · 2018Article
- Home infusion program with enzyme replacement therapy for Fabry disease: The experience of a large Italian collaborative group.Molecular genetics and metabolism reports · 2017Article
- Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naïve Fabry disease patients.BMC pharmacology & toxicology · 2017Observational
- Update on hypertrophic cardiomyopathy and a guide to the guidelines.Nature reviews. Cardiology · 2016Review
- Paediatric Fabry disease.Translational pediatrics · 2016Review
- Gastrointestinal Symptoms of Patients with Fabry Disease.Gastroenterology research and practice · 2016Article
- Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α.Genetics in medicine : official journal of the American College of Medical Genetics · 2014Observational
- Fabry disease: Evidence for a regional founder effect of theMolecular genetics and metabolism reports · 2014Article
- Cerebral hemodynamics and endothelial function in patients with Fabry disease.BMC neurology · 2013Article
- [Pain therapy for Fabry's disease].Der Internist · 2013Review
- Qualitative evaluation of the Canadian Fabry Disease Initiative.Canadian pharmacists journal : CPJ = Revue des pharmaciens du Canada : RPC · 2012Article
- Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy.Drug design, development and therapy · 2012Article
- Fabry disease: clinical and genotypic aspects of three cases in first degree relatives.Anais brasileiros de dermatologiaArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Fabry disease (FD) is an X-linked lysosomal storage disorder that affects both men and women. The manifestations of this heterogeneous disease are multisystemic and progressive. Prior to the development of enzyme replacement therapy, the management and treatment for Fabry disease was largely nonspecific and supportive. Because enzyme replacement therapy became commercially available in 2001, a variety of clinical benefits in Fabry patients have been consistently reported, including improved renal pathology and cardiac function, and reduced severity of neuropathic pain and improved pain-related quality of life. This update focuses on published data on the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa, and gives a brief overview on some of the outstanding management issues in the treatment of this complex disease.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.