Evidence map›Paper›PMID 42029427›Full record

ReviewJournal of neuromuscular diseases2026

TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACT.

Annemieke Aartsma-Rus, Laura Robertson, Lindsay N Alfano, Anna Mayhew, Heather Gordish-Dressman, Mike Kelly, Carl Morris, Volker Straub, Conrad C Weihl, Colin Werth and 4 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

14 authors.

Annemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.ORCID 0000-0003-1565-654X
Laura RobertsonTREAT-NMD, Newcastle upon Tyne, UK.
Lindsay N AlfanoNationwide Children's Hospital, Columbus OH, USA.
Anna MayhewThe John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Heather Gordish-DressmanChildrens National Hospital, Washington DC, USA.
Mike KellyCureDuchenne, Newport Beach, CA, USA.
Carl MorrisPHDLConsulting, Boston, MA, USA.
Volker StraubThe John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Conrad C WeihlDepartment of Neurology, Washington University School of Medicine, Saint Louis, Misssouri, USA.
Colin WerthParent project muscular dystrophy adult advisory committee (PAAC), Washington, DC, USA.
Olivier M DorchiesUniversity of Grenoble-Alpes, Laboratory of Fundamental and Applied Bionergetics (Inserm U1055), Grenoble, France.ORCID 0000-0002-6665-7887
Amanda RiviereGenethon, Evry, France.ORCID 0009-0004-8204-2980
Kanneboyina NagarajuDepartment of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Binghamton, NY, USA.
Kathryn R WagnerTREAT-NMD, Newcastle upon Tyne, UK.ORCID 0000-0002-5315-0278

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The TREAT-NMD Advisory Committee for Therapeutics (TACT) provides academic and industry applicants involved in therapy development in the neuromuscular disease (NMD) space with constructive, multidisciplinary expert advice in order to help derisk NMD drug development and avoid potential therapies entering clinical trials prematurely and to improve trial design. The ultimate goal of TACT is to reduce the risk of type 1 errors (concluding a treatment is effective, while it is not), type 2 errors (concluding a treatment is not effective, while it is), or risks of questions and regulatory rejections later on in development.TACT celebrated its 15

Indexed as

clinical trialsexpert groupneuromuscular diseasespatient involvementpreclinical research

Identifiers

PMID42029427
PMCPMC13438898

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.