Evidence map›Paper›PMID 26807415›Full record

ReviewAnnals of translational medicine2015

Niemann-Pick disease treatment: a systematic review of clinical trials.

Alejandro Santos-Lozano, Diana Villamandos García, Fabian Sanchis-Gomar, Carmen Fiuza-Luces, Helios Pareja-Galeano, Nuria Garatachea, Gisela Nogales Gadea, Alejandro Lucia

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Annals of translational medicine, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed
3.2field-weighted citation impact, top 8% of its field
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

33 citing papers in PubMed, 62 citations in OpenAlex.

  1. Review
  2. Observational
  3. Article
  4. Article
  5. Review
  6. Article
  7. Article
  8. Genetic Testing of Movements Disorders: A Review of Clinical Utility.Tremor and other hyperkinetic movements (New York, N.Y.) · 2024
    Review
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  10. A Novel SmallCells · 2023
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  11. Organ Weights inInternational journal of molecular sciences · 2022
    Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Article
  17. Article
  18. Article
  19. Insights into Lewy body disease from rare neurometabolic disorders.Journal of neural transmission (Vienna, Austria : 1996) · 2021
    Review
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 1 institution in 1 country.

Alejandro Santos-Lozano1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Diana Villamandos García1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Fabian Sanchis-Gomar1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Carmen Fiuza-Luces1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Helios Pareja-Galeano1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Nuria Garatachea1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Gisela Nogales Gadea1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Alejandro Lucia1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
Universidad de Zaragoza · ES

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The aim of this systematic review was to analyse all the published clinical trials assessing treatments for Niemann-Pick (NP) disease. At present there are only trials investigating the treatment of NP disease type C. Furthermore, there is no uniformity among studies in treatment outcomes or in data analysis and presentation of results. Miglustat is able to delay neurodegeneration, with greater benefits in patients with a late onset of the disease and β-cyclodextrin-hydroxypropyl (HBP-CD) can attenuate clinical symptoms. As for cholesterol-lowering drugs, the combination of lovastatin, cholestyramine and nicotinic acid is the most effective one for lowering cholesterolemia. Further research is much needed, and ongoing trials using enzyme replacement therapy might hopefully show promising results in the foreseeable future.

Indexed as

clinical trialmiglustatNiemann-Pick (NP) diseasetreatment

Identifiers

PMID26807415
PMCPMC4701532
OpenAlexW2246776136

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.