Evidence map›Paper›PMID 39598125›Full record

ArticleJournal of clinical medicine2024

The Unmet Needs of Lysosomal Storage Disorders from Early Diagnosis to Caregiving Pathways: An Italian Perspective.

Giancarlo Castaman, Silvia Linari, Antonio Barbato, Niko Costantino, Carlo Dionisi-Vici, Francesca Menni, Elena Procopio, Silvia Ramat, Fernanda Torquati, Elena Verrecchia and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of clinical medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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.

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.

  1. Article
  2. Neurodegenerative Diseases in Children: A Comprehensive Review.International journal of molecular sciences · 2026
    Review
  3. Observational
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

11 authors.

Giancarlo CastamanCenter for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, 50134 Florence, Italy.ORCID 0000-0003-4973-1317
Silvia LinariCenter for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, 50134 Florence, Italy.ORCID 0000-0002-5849-7377
Antonio BarbatoDepartment of Clinical Medicine and Surgery, "Federico II" University Hospital, 80131 Naples, Italy.ORCID 0000-0002-4660-821X
Niko CostantinoHead of Public Affairs, Cometa ASMME (Association for the Study of Inherited Metabolic Disorders), 35020 Padua, Italy.
Carlo Dionisi-ViciDivision of Metabolic Diseases and Hepatology, Ospedale Pediatrico Bambino Gesù IRCCS, 00165 Rome, Italy.ORCID 0000-0002-0007-3379
Francesca MenniFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Regional Clinical Center for Expanded Newborn Screening, 20122 Milan, Italy.ORCID 0000-0003-2724-2811
Elena ProcopioMeyer Children's Hospital IRCCS, 50139 Firenze, Italy.ORCID 0000-0002-8534-9951
Silvia RamatParkinson Unit, Neuromuscular-Skeletal and Sensory Organs Department, AOU Careggi, 50134 Firenze, Italy.ORCID 0000-0003-1301-9678
Fernanda TorquatiPresident of the Italian Gaucher Association and Member of the European Gaucher Alliance, 50066 Reggello, Italy.
Elena VerrecchiaPeriodic Fever and Rare Diseases Research Centre, Catholic University of Sacred Heart, 00168 Rome, Italy.ORCID 0000-0003-2131-2110
Maurizio ScarpaRegional Coordinator Centre for Rare Diseases, University Hospital of Udine, 33100 Udine, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

diagnostic delaylysosomal storage disordermultidisciplinaritynewborn screeningterritorial managementtransition

Identifiers

PMID39598125
PMCPMC11595360

What OpenQuestion holds

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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.