Evidence map›Paper›PMID 31344955›Full record

ArticleJournal of clinical medicine2019

Erythrocyte Encapsulated Thymidine Phosphorylase for the Treatment of Patients with Mitochondrial Neurogastrointestinal Encephalomyopathy: Study Protocol for a Multi-Centre, Multiple Dose, Open Label Trial.

Bridget E Bax, Michelle Levene, Murray D Bain, Lynette D Fairbanks, Massimiliano Filosto, Sema Kalkan Uçar, Thomas Klopstock, Cornelia Kornblum, Hanna Mandel, Shamima Rahman and 8 more

Open access · goldAbstract read
In one paragraph

Article in Journal of clinical medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed
2.8field-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

27 citing papers in PubMed, 48 citations in OpenAlex.

  1. Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleusMolecular therapy. Methods & clinical development · 2024
    Article
  2. Review
  3. Article
  4. Review
  5. Review
  6. A clinical approach to diagnosis and management of mitochondrial myopathies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
    Review
  7. Review
  8. Article
  9. Review
  10. Review
  11. Article
  12. Therapy Prospects for Mitochondrial DNA Maintenance Disorders.International journal of molecular sciences · 2021
    Review
  13. Article
  14. Review
  15. Article
  16. Moving towards clinical trials for mitochondrial diseases.Journal of inherited metabolic disease · 2021
    Review
  17. Article
  18. Article
  19. Pregnancy in MNGIE: a clinical and metabolic honeymoon.Annals of clinical and translational neurology · 2020
    Article
  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

18 authors at 10 institutions in 6 countries.

Bridget E BaxMolecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK. bebax@sgul.ac.uk.ORCID 0000-0001-6306-080X
Michelle LeveneMolecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK.
Murray D BainMolecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK.
Lynette D FairbanksThe Purine Research Laboratory, St Thomas' Hospital, London SE1 7EH, UK.
Massimiliano FilostoCentre for Neuromuscular Diseases, ASST Spedali Civili and University of Brescia, 25100 Brescia, Italy.ORCID 0000-0002-2852-7512
Sema Kalkan UçarDivision of Inborn Error of Metabolism, Ege University Medical Faculty, 35100 Izmir, Turkey.
Thomas KlopstockDepartment of Neurology, Friedrich-Baur-Institute, University of Munich, Ziemssenstr. 1, 80336 Munich, Germany.
Cornelia KornblumDepartment of Neurology, University Hospital Bonn, 53127 Bonn, Germany.
Hanna MandelGalilee Medical Center, Nahariya 22100, Israel.
Shamima RahmanMitochondrial Research Group, UCL London Great Ormond Street Institute of Child Health, London WC1N 1EH, UK.ORCID 0000-0003-2088-730X
Agathe RoubertieDepartment of Pediatric Neurology, Centre Hospitalier Universitaire de Montpellier, 34295 Montpellier, France.
Mauro ScarpelliInstitute of Neurology, University of Verona, 37126 Verona, Italy.
Philip M SedgwickInstitute for Medical and Biomedical Education, St George's, University of London, London SW17 0RE, UK.ORCID 0000-0001-8859-2175
Moshe BaruOrphan Technologies, Zürcherstrasse 19, 8640 Rapperswil, Switzerland.
Marcia Sellos-MouraOrphan Technologies, Zürcherstrasse 19, 8640 Rapperswil, Switzerland.
Jeanie PriceOrphan Technologies, Zürcherstrasse 19, 8640 Rapperswil, Switzerland.
Patrick HornAlbireo Pharma, Inc., Boston, MA 02109, USA.
Niranjanan NirmalananthanMolecular and Clinical Sciences, St. George's, University of London, London SW17 0RE, UK.
St George's, University of London · GBCentre Hospitalier Universitaire de Montpellier · FREge University · TRGerman Center for Neurodegenerative Diseases · DEGreat Ormond Street Hospital for Children NHS Foundation Trust · GBSt Thomas' Hospital · GBUniversity Hospital Bonn · DEUniversity of Brescia · ITUniversity of Verona · ITWestern Galilee Hospital · IL

Funding

Medical Research Council G0902179Medical Research Council MR/K025406/1Orphan Technologies MR/K025406/1
6 · The paper itself

Abstract

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder which primarily affects the gastrointestinal and nervous systems. This disease is caused by mutations in the nuclear

Indexed as

enzyme replacementerythrocyte encapsulated thymidine phosphorylasemitochondrial diseasemitochondrial neurogastrointestinal encephalomyopathyMNGIEmultiple doseorphan diseasePhase IIrare diseasethymidine phosphorylaseTYMP

Identifiers

PMID31344955
PMCPMC6722784
OpenAlexW2963717756

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.