ReviewJournal of nephrology2021
New drugs for the treatment of Anderson-Fabry disease.
Review in Journal of nephrology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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
11 citing papers in PubMed, 20 citations in OpenAlex.
- The effect of the glucosylceramide synthase inhibitor lucerastat on cardiac repolarization: results from a thorough QT study in healthy subjects.Orphanet journal of rare diseases · 2020Trial
- Unraveling A4GALT Mechanism and Its Modulation With Adamantyl-Galactosylceramide Analogues: Advancing Fabry Disease Therapeutic Strategies.Angewandte Chemie (International ed. in English) · 2026Article
- Review
- Polycystic kidney disease complicates renal pathology in a family with Fabry disease.Molecular genetics and metabolism reports · 2022Article
- Expert opinion on the recognition, diagnosis and management of children and adults with Fabry disease: a multidisciplinary Turkey perspective.Orphanet journal of rare diseases · 2022Review
- Chaperone Therapy in Fabry Disease.International journal of molecular sciences · 2022Review
- Fabry disease: what the cardiologist should consider in non-cardiac screening, diagnosis, and management-narrative review.Cardiovascular diagnosis and therapy · 2021Review
- New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy.Orphanet journal of rare diseases · 2021Review
- Renal Manifestations of Fabry Disease: A Narrative Review.Canadian journal of kidney health and disease · 2021Review
- Fabry Disease Therapy: State-of-the-Art and Current Challenges.International journal of molecular sciences · 2020Review
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Enzyme replacement therapy (ERT) of the Anderson-Fabry disease (AFD) has changed the outcome of patients. However, ERT has some limitations: a restricted volume of distribution, requirement for intravenous access, and stimulation of the production of anti-drug antibodies. Studies of new drugs aiming to improve the clinical effectiveness and convenience of therapy have been reported. Migalastat, a pharmacological chaperone, increases available enzymate activity in patients with mutations amenable to the therapy, is now available for clinical practice. It is orally administered, and while clinical trial results are promising, long term real world follow up is awaited. PEGylated enzyme has a longer half-life and potentially reduced antigenicity, compared with standard preparations; investigation of whether a longer dosing interval is viable is under way. Moss-derived enzyme has a higher affinity for mannose receptors, and appears to have access to renal tissue. Substrate reduction therapy is based on reducing the catabolism processes of the glycosphingolipids, and is currently under investigation as monotherapy. Gene therapy has now been initiated in clinical trail of in vivo and ex vivo technologies with early results are emerging. ERT represents a certain milestone of therapy for AFD with Migalastat now a newly available option. Other agents in clinical trial prevent further potential opportunities to improve outcomes in AFD.
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.