{rfName}
Ni

Indexed in

License and use

Altmetrics

Grant support

G Nogales-Gadea is supported by a Miguel Servet grant ISCIII CD14/00032, PI15/01756 and FEDER. Research studies by Alejandro Lucia are funded by the Fondo de Investigaciones Sanitarias (FIS, grant number PI12/00914) and cofinanced by Fondos FEDER.

Analysis of institutional authors

Pareja-Galeano, HeliosAuthor
Share
Publications
>
Review

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

Publicated to:Annals of Translational Medicine. 3 (22): 360- - 2015-12-01 3(22), DOI: 10.3978/j.issn.2305-5839.2015.12.04

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

Affiliations

‎ Autonomous Univ Barcelona, Badalona, Spain - Author
‎ Autonomous Univ Barcelona, Germans Trias & Pujol Res Inst, Neurosci Dept, Translat Res Lab Neuromuscular Dis, Badalona, Spain - Author
‎ European Univ Miguel de Cervantes, Dept Hlth Sci, GIDFYS, Valladolid, Spain - Author
‎ European Univ, Madrid, Spain - Author
‎ Hosp 12 Octubre I12, Res Inst, Madrid, Spain - Author
‎ Univ Leon, Fac Hlth Sci, Ponferrada, Spain - Author
‎ Univ Zaragoza, Dept Physiotherapy & Nursing, Huesca, Spain - Author
‎ Univ Zaragoza, GENUD Growth Exercise Nutr & Dev Res Grp, Zaragoza, Spain - Author
See more

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 beta-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.

Keywords
clinical trialmiglustatniemann-pick (np) diseaseClinical trialMiglustatNiemann-pick (np) diseaseTreatment

Quality index

Bibliometric impact. Analysis of the contribution and dissemination channel

The work has been published in the journal Annals of Translational Medicine due to its progression and the good impact it has achieved in recent years, according to the agency Scopus (SJR), it has become a reference in its field. In the year of publication of the work, 2015, it was in position , thus managing to position itself as a Q2 (Segundo Cuartil), in the category Medicine (Miscellaneous).

From a relative perspective, and based on the normalized impact indicator calculated from World Citations from Scopus Elsevier, it yields a value for the Field-Weighted Citation Impact from the Scopus agency: 3.77, which indicates that, compared to works in the same discipline and in the same year of publication, it ranks as a work cited above average. (source consulted: ESI Nov 14, 2024)

This information is reinforced by other indicators of the same type, which, although dynamic over time and dependent on the set of average global citations at the time of their calculation, consistently position the work at some point among the top 50% most cited in its field:

  • Field Citation Ratio (FCR) from Dimensions: 12.04 (source consulted: Dimensions Apr 2025)

Specifically, and according to different indexing agencies, this work has accumulated citations as of 2025-04-30, the following number of citations:

  • WoS: 39
  • Scopus: 52
  • Europe PMC: 27
  • OpenCitations: 37
Impact and social visibility

From the perspective of influence or social adoption, and based on metrics associated with mentions and interactions provided by agencies specializing in calculating the so-called "Alternative or Social Metrics," we can highlight as of 2025-04-30:

  • The use, from an academic perspective evidenced by the Altmetric agency indicator referring to aggregations made by the personal bibliographic manager Mendeley, gives us a total of: 119.
  • The use of this contribution in bookmarks, code forks, additions to favorite lists for recurrent reading, as well as general views, indicates that someone is using the publication as a basis for their current work. This may be a notable indicator of future more formal and academic citations. This claim is supported by the result of the "Capture" indicator, which yields a total of: 119 (PlumX).

With a more dissemination-oriented intent and targeting more general audiences, we can observe other more global scores such as:

  • The Total Score from Altmetric: 1.25.
  • The number of mentions on the social network X (formerly Twitter): 1 (Altmetric).