# Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial

**Type:** Papers  
**Canonical URL:** https://scholariq.org/papers/antisense-oligonucleotide-targeting-dmpk-in-patients-with-myotonic-dystrophy/

## Facts

| Field | Value |
| --- | --- |
| Author Names | Charles A. Thornton,Richard T. Moxley,Katy Eichinger,Chad Heatwole,Laurence Mignon,W. David Arnold,Tetsuo Ashizawa,John W Day,Gersham Dent,Matthew Tanner,Tina Duong,Ericka Greene,Laura Herbelin,Nicholas E. Johnson,Wendy King,John T. Kissel,Doris G. Leung,Donovan J. Lott,Daniel A Norris,Evan M Pucillo,W Schell,Jeffrey Statland,Nikia Stinson,S. H. Subramony,Shuting Xia,Kathie M. Bishop,C. Frank Bennett |
| Citations | 62 |
| DOI | 10.1016/s1474-4422(23)00001-7 |
| Fields | Biochemistry, Genetics and Molecular Biology,Medicine,Neuroscience |
| Open Access | false |
| OA Status | closed |
| OpenAlex ID | https://openalex.org/W4320888593 |
| PMID | 36804094 |
| Type | article |
| Year | 2023 |

## Paper authors

- [Laurence Mignon](https://scholariq.org/researchers/laurence-mignon/)

## Paper primary topic

- [Genetic Neurodegenerative Diseases](https://scholariq.org/topics/genetic-neurodegenerative-diseases/)

## Paper topics

- [Genetic Neurodegenerative Diseases](https://scholariq.org/topics/genetic-neurodegenerative-diseases/)
- [Biochemical and Molecular Research](https://scholariq.org/topics/biochemical-and-molecular-research/)
- [Amyotrophic Lateral Sclerosis Research](https://scholariq.org/topics/amyotrophic-lateral-sclerosis-research/)

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Source: ScholarIQ — public research metadata, principally OpenAlex. See https://scholariq.org/sources/ for provenance and https://scholariq.org/methodology/ for what these figures mean.