Evidence map›Paper›PMID 42074270›Full record

ArticleInternational journal of molecular sciences2026

Development of Small-Molecule Allosteric Modulators of Beta-Galactosidase (β-Gal) for the Treatment of GM1 Gangliosidosis and Morquio B.

Natàlia Pérez-Carmona, Elena Cubero, Ana Ruano, Maria Pons-Vizcarra, Aida Delgado, Ana Trapero, Marc Reves, Laura Rodríguez-Pascau, Joanne Taylor, Marc Martinell and 2 more

Abstract read
In one paragraph

Article in International journal of molecular sciences, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

12 authors.

Natàlia Pérez-CarmonaGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.
Elena CuberoGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0002-2239-6670
Ana RuanoGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.
Maria Pons-VizcarraGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0001-5008-5741
Aida DelgadoGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0003-2793-1545
Ana TraperoGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0003-4526-7895
Marc RevesMinoryx Therapeutics, 08302 Barcelona, Spain.
Laura Rodríguez-PascauMinoryx Therapeutics, 08302 Barcelona, Spain.ORCID 0000-0001-5631-1172
Joanne TaylorGain Therapeutics, Inc., Bethesda, MD 20814, USA.
Marc MartinellMinoryx Therapeutics, 08302 Barcelona, Spain.
Xavier BarrilGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0000-0002-0281-1347
Ana María García-CollazoGain Therapeutics Sucursal en España, Parc Científic de Barcelona, 08028 Barcelona, Spain.ORCID 0009-0004-9582-7429

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

GM1 gangliosidosis and Morquio B are rare lysosomal storage disorders (LSDs) with significant unmet medical needs. These disorders result from mutations in the galactosidase beta 1 (

Indexed as

beta-GalactosidaseGangliosidosis, GM1Mucopolysaccharidosis IVSmall Molecule LibrariesAllosteric RegulationAllosteric SiteAnimalsDogsFibroblastsHumansStructure-Activity Relationshipbeta-GalactosidaseSmall Molecule Librariesbeta-galactosidaseGLB1GM1-gangliosidosislysosomal storage disordersMagellan platformpharmacological chaperoneprotein misfoldingsite-directed enzyme enhancement therapy (SEE-Tx)structurally targeted allosteric regulators (STARs)substrate depletion

Identifiers

PMID42074270
PMCPMC13115887

What OpenQuestion holds

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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.