Evidence map›Paper›PMID 30337566›Full record

ArticleNature reviews. Disease primers2018

Author Correction: Lysosomal storage diseases.

Frances M Platt, Alessandra d'Azzo, Beverly L Davidson, Elizabeth F Neufeld, Cynthia J Tifft

Registry-linked trialAbstract readPublished Erratum
PubMed Publisher
In one paragraph

Article in Nature reviews. Disease primers, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04532047 (PEARL), which is not on this map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
–field-weighted citation impact
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.

NCT04532047 phase1recruitingnot on this mapstarted 2021, after this paper: background citation

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

TypeinterventionalSponsorUniversity of California, San FranciscoRan2021 to 2032Enrolled10ConditionsMPS I, MPS II, MPS IVA, MPS VIArmsAldurazyme (laronidase)
3 · Its place in the literature

Who cites it

8 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Organelle-Targeting Nanoparticles.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  5. Review
  6. Article
  7. Review
  8. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

5 authors.

Frances M PlattDepartment of Pharmacology, University of Oxford, Oxford, UK. frances.platt@pharm.ox.ac.uk.
Alessandra d'AzzoDepartment of Genetics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Beverly L DavidsonThe Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Elizabeth F NeufeldDepartment of Biological Chemistry, David Geffen School of Medicine, University of California-Los Angeles, Los Angeles, CA, USA.
Cynthia J TifftOffice of the Clinical Director and Medical Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In the version of the article originally published, in Figure 2 and the accompanying legend, LIMP 2 was incorrectly referred to as LIMP 1. The article has now been corrected.

Identifiers

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.