Evidence map›Paper›PMID 39287071›Full record

SynthesisJournal of comparative effectiveness research2024

Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.

Simon Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor, Alasdair MacCulloch, Neil Johnson, Jeff Castelli, Ilona Czarny-Ozga, Tahseen Mozaffar, Howard Thom

Erratum issuedAbstract readSystematic ReviewComparative StudyNetwork Meta-Analysis
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Journal of comparative effectiveness research, 2024. 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 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
–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

6 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Review
  5. Article
  6. Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases.American journal of medical genetics. Part C, Seminars in medical genetics · 2025
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors.

Simon ShohetAmicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.ORCID 0009-0003-0901-5233
Noemi HummelCertara GmbH, Chester Platz 1, 79539, Lörrach, Germany.
Shuai FuCertara, Office 610, South Tower, Hong Kong Plaza, No. 283 Huaihai Road Middle, Huangpu District, Shanghai, China.ORCID 0000-0003-1889-3030
Ian KeyzorAmicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.ORCID 0000-0001-5297-0816
Alasdair MacCullochAmicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.
Neil JohnsonAmicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK.
Jeff CastelliAmicus Therapeutics, Inc., 47 Hulfish St., Princeton, NJ, 08542, USA.
Ilona Czarny-OzgaCertara, Instytut Arcana sp. z o.o. 17, Kuklinskiego Street, 30-720, Krakow, Poland.
Tahseen MozaffarDepartment of Neurology, University of California, 1001 Health Sciences Road, Irvine, CA 92697-3950, USA.ORCID 0000-0002-1230-0188
Howard ThomPopulation Health Sciences, Bristol Medical School, University of Bristol, 1-5 Whiteladies Rd, Bristol, BS8 1NU, UK.ORCID 0000-0001-8576-5552

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

1-DeoxynojirimycinEnzyme Replacement TherapyGlycogen Storage Disease Type IIalpha-GlucosidasesHumansRandomized Controlled Trials as TopicWalk Test1-Deoxynojirimycinalpha-GlucosidasesGAA protein, humanmiglustatcipaglucosidase alfaindirect treatment comparisonmiglustatPompe disease

Identifiers

PMID39287071
PMCPMC11426283

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.