Evidence map›Paper›PMID 42137289›Full record

ArticleMolecular therapy. Advances2026

CSF GAG non-reducing ends in MPS IH after peripheral and brain-penetrating therapies: A model comparing IV non-targeted ERT and HCT.

Troy C Lund, John E Wagner, Susie Long, Ashish O Gupta, Paul J Orchard, Elizabeth Braunlin, Chester B Whitley, Julie B Eisengart

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 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

8 authors.

Troy C LundDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Minneapolis, MN 55455, USA.
John E WagnerDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Minneapolis, MN 55455, USA.
Susie LongDepartment of Pharmacy, Fairview MHealth, Minneapolis, MN 55455, USA.
Ashish O GuptaDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Minneapolis, MN 55455, USA.
Paul J OrchardDepartment of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Minneapolis, MN 55455, USA.
Elizabeth BraunlinDepartment of Pediatrics, Division of Pediatric Cardiology, University of Minnesota, Minneapolis, MN 55455, USA.
Chester B WhitleyAdvanced Therapies Program, University of Minnesota, Minneapolis, MN 55455, USA.
Julie B EisengartDepartment of Pediatrics, Division of Clinical Behavioral Neuroscience, University of Minnesota, Minneapolis, MN 55455, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucopolysaccharidosis type IH (MPS IH) is a lysosomal disease caused by insufficient L-iduronidase (IDUA), resulting in progressive accumulation of glycosaminoglycans (GAGs) in the central nervous system (CNS). Hematopoietic cell transplantation (HCT) replaces IDUA through cellular cross-correction, stabilizing the CNS. Intravenous (i.v.) enzyme replacement therapy (ERT) is also effective at reducing GAG accumulation; however, it is thought to inefficiently cross the blood-brain barrier. To compare the effect of i.v. ERT on GAG degradation in the CNS with the effect of brain-penetrant therapy, i.e., HCT, we measured cerebrospinal fluid (CSF) GAG non-reducing ends in patients with MPS IH who were ERT-naive (

Indexed as

biomarkersblood brain barrierenzyme replacement therapyhematopoietic stem cell transplantheparan sulfateHurler syndromemucopolysaccharidosis

Identifiers

PMID42137289
PMCPMC13148921

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Registered trials

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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.