ArticleBrain sciences2018
Best Practices in Fragile X Syndrome Treatment Development.
Article in Brain sciences, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07654114 (Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With Fragile X Syndrome), which is not on this map. Cited by 30 papers.
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.
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.
Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With Fragile X Syndrome
Who cites it
30 citing papers in PubMed.
- Effects of AFQ056 on language learning in fragile X syndrome.The Journal of clinical investigation · 2023Trial
- Baclofen-associated neurophysiologic target engagement across species in fragile X syndrome.Journal of neurodevelopmental disorders · 2022Trial
- Telehealth-enabled behavioral treatment for problem behaviors in boys with fragile X syndrome: a randomized controlled trial.Journal of neurodevelopmental disorders · 2020Trial
- Article
- Alpha oscillations are dysrhythmic in Fragile X syndrome.bioRxiv : the preprint server for biology · 2026Article
- Dysfunctional neural dynamics associated with sensory phenotypes in Fragile X syndrome: insights from mouse models.Journal of neurodevelopmental disorders · 2025Review
- Caregiver-reported meaningful change in functional domains for individuals with developmental and epileptic encephalopathy: A convergent mixed-methods design.Developmental medicine and child neurology · 2025Article
- FX ENTRAIN: scientific context, study design, and biomarker driven brain-computer interfaces in neurodevelopmental conditions.Frontiers in neuroscience · 2025Article
- Article
- What does better look like in individuals with severe neurodevelopmental impairments? A qualitative descriptive study on SCN2A-related developmental and epileptic encephalopathy.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2024Article
- Oral language interventions can improve language outcomes in children with neurodevelopmental disorders: A systematic review and meta-analysis.Campbell systematic reviews · 2023Review
- Reliability of resting-state electrophysiology in fragile X syndrome.Biomarkers in neuropsychiatry · 2023Article
- Sex Differences in Brain Disorders.International journal of molecular sciences · 2023Review
- Efficient Delivery ofGenes · 2023Article
- Hemispheric Utilization of Alpha Oscillatory Dynamics as a Unique Biomarker of Neural Compensation in Females with Fragile X Syndrome.ACS chemical neuroscience · 2022Article
- Long-term follow-up of telehealth-enabled behavioral treatment for challenging behaviors in boys with fragile X syndrome.Journal of neurodevelopmental disorders · 2022Article
- Improving clinical trial readiness to accelerate development of new therapeutics for Rett syndrome.Orphanet journal of rare diseases · 2022Review
- Article
- The Use of Peptides in the Treatment of Fragile X Syndrome: Challenges and Opportunities.Frontiers in psychiatry · 2021Article
- Gaboxadol in Fragile X Syndrome: A 12-Week Randomized, Double-Blind, Parallel-Group, Phase 2a Study.Frontiers in pharmacology · 2021Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Preclinical studies using animal models of fragile X syndrome have yielded several agents that rescue a wide variety of phenotypes. However, translation of these treatments to humans with the disorder has not yet been successful, shedding light on a variety of limitations with both animal models and human trial design. As members of the Clinical Trials Committee of the National Fragile X Foundation, we have discussed a variety of recommendations at the level of preclinical development, transition from preclinical to human projects, family involvement, and multi-site trial planning. Our recommendations are made with the vision that effective new treatment will lie at the intersection of innovation, rigorous and reproducible research, and stakeholder involvement.
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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.