ReviewDisease models & mechanisms2020
Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT).
Review in Disease models & mechanisms, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
What it found
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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.
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.
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Who cites it
17 citing papers in PubMed, 25 citations in OpenAlex.
- TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACT.Journal of neuromuscular diseases · 2026Review
- From Symptomatic Therapies to Disease-Modifying Approaches for Neuronal Sodium Channel Disorders.International journal of molecular sciences · 2025Review
- A two-site collaborative study of electrical impedance myography for evaluation of disease progression in murine Duchenne muscular dystrophy models.Scientific reports · 2025Article
- Teaming up to overcome challenges toward translation of new therapeutics for osteoarthritis.Journal of orthopaedic research : official publication of the Orthopaedic Research Society · 2024Article
- Branched-Chain Amino Acids and Di-Alanine Supplementation in Aged Mice: A Translational Study on Sarcopenia.Nutrients · 2023Article
- Current knowledge, challenges and innovations in developmental pharmacology: A combined conect4children Expert Group and European Society for Developmental, Perinatal and Paediatric Pharmacology White Paper.British journal of clinical pharmacology · 2022Review
- Planning for progress: A US regulatory approach to advancing the clinical development of gene therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Natural history study and statistical modeling of disease progression in a preclinical model of myotubular myopathy.Disease models & mechanisms · 2022Article
- The predictive value of models of neuromuscular disorders to potentiate clinical translation.Disease models & mechanisms · 2022Article
- FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?Journal of personalized medicine · 2022Review
- The Role of Patient Involvement When Developing Therapies.Nucleic acid therapeutics · 2022Article
- β-Dystroglycan Restoration and Pathology Progression in the DystrophicBiomolecules · 2021Article
- Using the concept of "deserved trust" to strengthen the value and integrity of biomedical research.Accountability in research · 2021Article
- Do we really know how many clinical trials are conducted ethically? Why research ethics committee review practices need to be strengthened and initial steps we could take to strengthen them.Journal of medical ethics · 2021Review
- Moving towards less biased research.BMJ open science · 2021Article
- Young-Onset Carcinogenesis - The Potential Impact of Perinatal and Early Life Metabolic Influences on the Epigenome.Frontiers in oncology · 2021Article
- Moving neuromuscular disorders research forward: from novel models to clinical studies.Disease models & mechanisms · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 7 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden for the whole disease community. Translation of data from preclinical studies to justify any clinical trial must be carefully pondered in order to minimize the risk of clinical trial withdrawal or failure. A rigorous distinction between proof-of-concept and preclinical efficacy studies using animal models is key to support the rationale of a clinical trial involving patients. This Review evaluates the experience accumulated by the TREAT-NMD Advisory Committee for Therapeutics, which provides detailed constructive feedback on clinical proposals for neuromuscular diseases submitted by researchers in both academia and industry, and emphasizes that a timely critical review of preclinical efficacy data from animal models, including biomarkers for specific diseases, combined with adherence to existing guidelines and standard protocols, can significantly help to de-risk clinical programs and prevent disappointments and costly engagement.
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What OpenQuestion holds
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.