Evidence map›Paper›PMID 40255904›Full record

ArticleMolecular therapy. Nucleic acids2025

Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease.

Ryan A Oliver, Meghan E Ahern, Perla G Castaneda, Tushare Jinadasa, Anirban Bardhan, Kathy Y Morgan, Kristin Ha, Kailash Adhikari, Nino Jungels, Noa Liberman and 13 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 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

23 authors.

Ryan A OliverSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Meghan E AhernSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Perla G CastanedaSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Tushare JinadasaSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Anirban BardhanSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Kathy Y MorganSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Kristin HaSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Kailash AdhikariSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Nino JungelsSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Noa LibermanSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Anindita MitraSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Christopher D GreerSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Alec M WrightSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Emily G ThompsonSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Stephanie GarciaSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Elena CopsonSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Senkara AlluSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Xuyu TanSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Alex J CallahanSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Bao Zhong CaiSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Vincent GuerlavaisSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Kevin J KimSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.
Annika B MalmbergSarepta Therapeutics, Inc, 215 First Street, Cambridge, MA 02142, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Late-onset Pompe disease (LOPD) is overwhelmingly caused by a single mutation that disrupts splicing of acid-alpha glucosidase (

Indexed as

late-onset Pompe diseaselysosomal storage diseaseMT: Oligonucleotides: Therapies and Applicationsoligonucleotide therapeuticsRNA medicinessplice switching antisense oligonucleotide

Identifiers

PMID40255904
PMCPMC12008586

What OpenQuestion holds

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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.