Evidence map›Paper›PMID 40060931›Full record

ArticleMolecular therapy. Nucleic acids2025

Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S.

Sandra Smieszek, Bartlomiej Przychodzen, Christina Tyner, Caroline Johnson, Haimeng Bai, Jennifer M Kwon, D Walker Hagan, Caitlyn Niccum, Rocky Brighton, Kenneth Hawkins and 7 more

Registry-linked trialAbstract 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. It is linked to trial NCT07223632 (The Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S), which is not on this map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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.

NCT07223632 phase1 / phase2active not recruitingnot on this map

The Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient With Confirmed IGHMBP2 Intronic Cryptic Splice Variant C. 1235+894C>A That is Amenable to Antisense Oligonucleotide (ASO)-Mediated Correction of IGHMBP2 Splicing

TypeinterventionalSponsorVanda PharmaceuticalsRan2025 to 2027Enrolled1ConditionsCharcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)ArmsVCA-894A
3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Article
  2. Review
  3. Junctions in Jeopardy: the neuromuscular junction is a selective pathological target in Charcot-Marie-Tooth disease.Mammalian genome : official journal of the International Mammalian Genome Society · 2026
    Review
  4. A class of deep intronicmedRxiv : the preprint server for health sciences · 2026
    Article
  5. Article
  6. 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

17 authors.

Sandra SmieszekVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Bartlomiej PrzychodzenVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Christina TynerVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Caroline JohnsonVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Haimeng BaiVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Jennifer M KwonDepartment of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI 53705, USA.
D Walker HaganHesperos, Inc., Orlando, FL 32826, USA.
Caitlyn NiccumHesperos, Inc., Orlando, FL 32826, USA.
Rocky BrightonHesperos, Inc., Orlando, FL 32826, USA.
Kenneth HawkinsUniversity of Central Florida, NanoScience Technology Center, Orlando, FL 32826, USA.
Romy AikenUniversity of Central Florida, NanoScience Technology Center, Orlando, FL 32826, USA.
Ahmad NawazUniversity of Central Florida, NanoScience Technology Center, Orlando, FL 32826, USA.
Xiufang GuoUniversity of Central Florida, NanoScience Technology Center, Orlando, FL 32826, USA.
James HickmanHesperos, Inc., Orlando, FL 32826, USA.
Christos M PolymeropoulosVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Gunther BirznieksVanda Pharmaceuticals Inc., Washington, DC 20037, USA.
Mihael H PolymeropoulosVanda Pharmaceuticals Inc., Washington, DC 20037, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Immunoglobulin mu-binding protein 2 (

Indexed as

antisense oligonucleotideCharcot-Marie-Tooth Disease type 2Sexon skippinggenetic rescueIGHMBP2MT: Oligonucleotides: Therapies and Applicationspersonalized treatmentRNA therapeuticssplicing variants

Identifiers

PMID40060931
PMCPMC11889396

What OpenQuestion holds

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Registered trials

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.