Evidence map›Paper›PMID 29487322›Full record

ArticleScientific reports2018

Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1.

Elina Makino, Helen Klodnitsky, John Leonard, James Lillie, Troy C Lund, John Marshall, Jennifer Nietupski, Paul J Orchard, Weston P Miller, Clifford Phaneuf and 4 more

Erratum issuedOpen access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Scientific reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.3field-weighted citation impact, top 42% 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

3 citing papers in PubMed, 4 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

14 authors at 2 institutions in 1 country.

Elina MakinoDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA. elina.makino@sanofi.com.
Helen KlodnitskyDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
John LeonardRare Disease, R&D, Sanofi, Framingham, MA, 01701, USA.
James LillieDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
Troy C LundDivision of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, 55455, USA.
John MarshallRare Disease, R&D, Sanofi, Framingham, MA, 01701, USA.
Jennifer NietupskiRare Disease, R&D, Sanofi, Framingham, MA, 01701, USA.
Paul J OrchardDivision of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, 55455, USA.
Weston P MillerDivision of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, 55455, USA.
Clifford PhaneufDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
Drew TietzDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
Mariet L VarbanDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
Marissa DonovanDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
Alexey BelenkiDrug Discovery, R&D, Sanofi, Waltham, MA, 02451, USA.
Sanofi (United States) · USUniversity of Minnesota · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Certain recessively inherited diseases result from an enzyme deficiency within lysosomes. In mucopolysaccharidoses (MPS), a defect in glycosaminoglycan (GAG) degradation leads to GAG accumulation followed by progressive organ and multiple system dysfunctions. Current methods of GAG analysis used to diagnose and monitor the diseases lack sensitivity and throughput. Here we report a LC-MS method with accurate metabolite mass analysis for identifying and quantifying biomarkers for MPS type I without the need for extensive sample preparation. The method revealed 225 LC-MS features that were >1000-fold enriched in urine, plasma and tissue extracts from untreated MPS I mice compared to MPS I mice treated with iduronidase to correct the disorder. Levels of several trisaccharides were elevated >10000-fold. To validate the clinical relevance of our method, we confirmed the presence of these biomarkers in urine, plasma and cerebrospinal fluid from MPS I patients and assessed changes in their levels after treatment.

Indexed as

AnimalsBiomarkersChromatography, LiquidDisease Models, AnimalFemaleGlycosaminoglycansHeparan SulfateHumansIduronidaseMaleMiceMucopolysaccharidosis ITrisaccharidesBiomarkersGlycosaminoglycansHeparan SulfateIduronidaseTrisaccharides

Identifiers

PMID29487322
PMCPMC5829143
OpenAlexW2796270994

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.