Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patients.

Rahul Soangra, Marybeth Grant-Beuttler, Harriet Chang, Raymond Y Wang

Abstract read

In one paragraph

Article in Scientific reports, 2025. 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

4 authors.

Rahul SoangraCrean College of Health and Behavioral Sciences, Chapman University, Orange, CA, 92866, USA.

Marybeth Grant-Beuttler *Department of Physical Therapy, Oregon Institute of Technology, Klamath Falls, OR, 97601, USA.

Harriet Chang *Division of Metabolic Disorders, CHOC Children's Hospital, Orange, CA, 92868, USA.

Raymond Y WangDivision of Metabolic Disorders, CHOC Children's Hospital, Orange, CA, 92868, USA. RaWang@choc.org.

Funding

AI-based Fall-Risk Assessment during Daily Activities in Post Stroke Survivors using SmartphonesR15HD110941 · NICHD · CHAPMAN UNIVERSITY · PI SOANGRA, RAHUL · 2023 to 2023

$391k

Eunice Kennedy Shriver National Institute of Child Health and Human Development 1R15HD110941-01NICHD NIH HHS R15 HD110941

6 · The paper itself

Abstract

Neuronal ceroid lipofuscinosis type 2 (CLN2) is a rapidly progressive neurodegenerative disorder leading to premature mortality. Ambulatory CLN2 patients typically receive standard of care treatment through biweekly intracerebroventricular (ICV) enzyme replacement therapy (ERT) involving recombinant human tripeptidyl peptidase 1, known as cerliponase alfa (Brineura

Indexed as

AminopeptidasesDipeptidyl-Peptidases and Tripeptidyl-PeptidasesEnzyme Replacement TherapyGaitNeuronal Ceroid-LipofuscinosesPostural BalanceTripeptidyl-Peptidase 1AdolescentFemaleFollow-Up StudiesHumansLongitudinal StudiesMaleRecombinant ProteinsSerine ProteasesAminopeptidasescerliponase alfaDipeptidyl-Peptidases and Tripeptidyl-PeptidasesRecombinant ProteinsSerine ProteasesTPP1 protein, humanTripeptidyl-Peptidase 1

Identifiers

PMID39762319

DOI10.1038/s41598-024-82157-5

PMCPMC11704126

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.