Trial reportGraefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie2009
Does enzyme replacement therapy influence the ocular changes in type VI mucopolysaccharidosis?
Trial report in Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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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
16 citing papers in PubMed, 1 synthesis or guideline pooled it, 48 citations in OpenAlex.
- Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.The Cochrane database of systematic reviews · 2021Pooled it
- Consensus-based expert recommendations on the management of MPS IVa and VI in Saudi Arabia.Orphanet journal of rare diseases · 2024Article
- Mucopolysaccharidosis: A broad review.Indian journal of ophthalmology · 2022Review
- Management of Corneal Clouding in Patients with Mucopolysaccharidosis.Journal of clinical medicine · 2021Review
- Progression of eye disease over 15 years in a patient with mucopolysaccharidosis type VI on enzyme replacement therapy.BMJ case reports · 2021Article
- Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.International journal of molecular sciences · 2020Review
- Ophthalmologic manifestations in Taiwanese patients with mucopolysaccharidoses.Molecular genetics & genomic medicine · 2019Article
- Fundoscopic Changes in Maroteaux-Lamy Syndrome.Case reports in ophthalmological medicine · 2019Article
- Enzyme replacement therapy: efficacy and limitations.Italian journal of pediatrics · 2018Review
- Lacritin and other autophagy associated proteins in ocular surface health.Experimental eye research · 2016Review
- Ocular manifestations and management recommendations of lysosomal storage disorders I: mucopolysaccharidoses.Clinical ophthalmology (Auckland, N.Z.) · 2015Review
- Retinal degeneration in mucopolysaccharidose type II.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2013Article
- Visual Dysfunction of Type I and VI Mucopolysaccharidosis Patients Evaluated with Visual Evoked Cortical Potential.Case reports in ophthalmology · 2012Article
- Mucopolysaccharidoses in northern Brazil: Targeted mutation screening and urinary glycosaminoglycan excretion in patients undergoing enzyme replacement therapy.Genetics and molecular biology · 2011Article
- Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Clinical ocular manifestations of Taiwanese patients with mucopolysaccharidoses VI (Maroteaux-Lamy syndrome).Taiwan journal of ophthalmologyArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTo describe the ocular changes noted in seven patients with type VI mucopolysaccharidosis (MPS VI) during 44 months of follow-up while on enzyme replacement therapy (ERT).
methodsOne male and six female patients with MPS VI were followed-up for a mean period of 44 months while undergoing enzyme replacement therapy (ERT) with recombinant arylsulfatase B (Naglazyme). They were examined annually for visual acuity, corneal clouding, intraocular pressure (IOP), optic nerve head and fundus morphology. Corneal clouding was documented by photography. We acknowledge that our methodology may not have been sensitive enough to detect extremely mild ocular changes, including minimal increases in corneal thickness or clouding. Nevertheless, this limitation has been considered in the interpretation of our findings.
resultsOphthalmological findings remained stable in 5/7 patients. One patient experienced a modest improvement in visual acuity of more than 2 Snellen lines in one eye, while another patient suffered a deterioration in visual acuity of more than 2 Snellen lines in both eyes. Five out of seven patients showed optic nerve pathology: two of these exhibited optic nerve head swelling, while the other three showed variable degrees of optic nerve atrophy. All seven patients suffered from the typical corneal stromal opacities, however, to variable extents.
conclusionVisual function and ocular findings did not deteriorate in six out of seven MPS VI patients during a mean follow-up period of 3 and a half years on ERT.
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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.