ArticleMedicine and pharmacy reports2021
Gaucher disease - therapeutic aspects in Romania.
Article in Medicine and pharmacy reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.
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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.
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
1 citing paper in PubMed, 1 synthesis or guideline pooled it, 1 citations in OpenAlex.
- Gaucher: A Systematic Review on Oral and Radiological Aspects.Medicina (Kaunas, Lithuania) · 2023Pooled it
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gaucher disease is a rare autosomal recessive disease caused by the beta-glicosidase activity deficiency, which will lead to substrate accumulation mainly in the liver, spleen or bone marrow. The main symptoms are liver and spleen enlargement, anemia and low platelet count, bone crisis and fatigue. Several treatment options are available, as enzyme replacement therapy, substrate reduction therapy, or chaperones treatment whose effect is still studied. There are 77 adult patients treated at this time in Romania, 54 with intravenous enzyme replacement ant 23 with oral substrate reduction therapy. No severe adverse effects have been reported by now. All patients had improved disease related symptoms after the receiving of the treatment.
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Registered trials
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