Evidence map›Paper›PMID 37927484›Full record

ArticleJIMD reports2023

Comparison of efficacy between subcutaneous and intravenous application of moss-aGal in the mouse model of Fabry disease.

Paulina Dabrowska-Schlepp, Andreas Busch, Jin-Song Shen, Rachel Y Cheong, Lone Bruhn Madsen, Daniel Mascher, Raphael Schiffmann, Andreas Schaaf

Open access · goldAbstract read
In one paragraph

Article in JIMD reports, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
0.5field-weighted citation impact, top 34% 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

5 citing papers in PubMed, 1 synthesis or guideline pooled it, 3 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Article
  4. Review
  5. 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

8 authors at 4 institutions in 4 countries.

Paulina Dabrowska-SchleppEleva GmbH Freiburg Germany.ORCID https://orcid.org/0000-0002-9906-667X
Andreas BuschEleva GmbH Freiburg Germany.
Jin-Song ShenInstitute of Metabolic Disease, Baylor Scott & White Research Institute Dallas Texas USA.
Rachel Y CheongScantox Sweden (Previously Timeline Bioresearch AB) Lund Sweden.ORCID https://orcid.org/0000-0003-0264-7636
Lone Bruhn MadsenScantox Sweden (Previously Timeline Bioresearch AB) Lund Sweden.ORCID https://orcid.org/0009-0003-2069-1849
Daniel Mascherpharm-analyt Labor GmbH Baden Austria.
Raphael SchiffmannInstitute of Metabolic Disease, Baylor Scott & White Research Institute Dallas Texas USA.ORCID https://orcid.org/0000-0003-2437-0252
Andreas SchaafEleva GmbH Freiburg Germany.
University of Education Freiburg · DEBaylor Scott & White Health · USBiocrine (Sweden) · SEIonicon Analytik (Austria) · AT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Fabry disease (FD, OMIM 301500) is a rare X-linked inherited lysosomal storage disorder associated with reduced activities of α-galactosidase A (aGal, EC 3.2.1.22). The current standard of care for FD is based on enzyme replacement therapy (ERT), in which a recombinantly produced version of αGal is intravenously (iv) applied to Fabry patients in biweekly intervals. Though the iv application is clinically efficacious, periodical infusions are inconvenient, time- and resource-consuming and they negatively impact the patients' quality of life. Subcutaneous (sc) injection, in contrast, is an established route of administration for treatment of chronic conditions. It opens the beneficial option of self-administration, thereby improving patients' quality of life and at the same time reducing treatment costs. We have previously shown that Moss-α-Galactosidase (moss-aGal), recombinantly produced in the moss

Indexed as

enzyme replacement therapyFabry diseasemoss‐aGalsubcutaneous application

Identifiers

PMID37927484
PMCPMC10623099
OpenAlexW4386695438

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.