ArticleBrain sciences2019
Voice of People with Fragile X Syndrome and Their Families: Reports from a Survey on Treatment Priorities.
Article in Brain sciences, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers, 2 of them syntheses that pooled 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.
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.
Who cites it
36 citing papers in PubMed, 2 syntheses or guidelines pooled it, 57 citations in OpenAlex.
- The Impact of Fragile X Syndrome on Caregivers: A Systematic Review.Journal of intellectual disability research : JIDR · 2026Pooled it
- Psychiatric difficulties in females with fragile X syndrome: a systematic review and meta-analyses.Frontiers in psychiatry · 2026Pooled it
- Parent-Reported Outcome Measures for Individuals with Fragile X Syndrome: Clinically Meaningful Change Thresholds.Journal of autism and developmental disorders · 2026Trial
- ROC Analysis of Biomarker Combinations in Fragile X Syndrome-Specific Clinical Trials: Evaluating Treatment Efficacy via Exploratory Biomarkers.Translational psychiatry · 2025Trial
- Effects of AFQ056 on language learning in fragile X syndrome.The Journal of clinical investigation · 2023Trial
- A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome.Journal of neurodevelopmental disorders · 2019Trial
- Profile of Behavioral Comorbidities in Children With Fragile X Syndrome.Journal of autism and developmental disorders · 2026Article
- Challenging behaviors across COVID-19 in young children with rare neurogenetic conditions: A seven-year, cross-syndrome analysis.Child development · 2026Article
- Caregiver-perceived behavioral challenges in fragile X syndrome and implications for measuring treatment benefit in clinical trials.Journal of patient-reported outcomes · 2026Article
- Blarcamesine improves EEG biomarkers of cortical dysfunction in a mouse model of fragile X syndrome.Frontiers in pharmacology · 2026Article
- Gene Therapy for Fragile X Syndrome, Challenges, and Promises.The journal of gene medicine · 2025Review
- Relationship Between Intellectual Disability and Behavioral Comorbidity in Children With Fragile X Syndrome.Journal of autism and developmental disorders · 2025Article
- The Aberrant Behavior Checklist for Fragile X Syndrome: A Qualitative Clinician Evaluation of Content Validity.Journal of child and adolescent psychopharmacology · 2025Article
- Preliminary perspectives on gene therapy in fragile X syndrome: a caregiver view.Journal of neurodevelopmental disorders · 2025Article
- Auditory N1 event-related potential amplitude is predictive of serum concentration of BPN14770 in fragile X syndrome.Molecular autism · 2024Article
- Fragile X Syndrome and FMR1 premutation: results from a survey on associated conditions and treatment priorities in Italy.Orphanet journal of rare diseases · 2024Article
- Auditory N1 event-related potential amplitude is predictive of serum concentration of BPN14770 in fragile x syndrome.Research square · 2024Article
- Challenges in developing therapies in fragile X syndrome: how the FXLEARN trial can guide research.The Journal of clinical investigation · 2024Article
- Latent Class Analysis Identifies Distinctive Behavioral Subtypes in Children with Fragile X Syndrome.Journal of autism and developmental disorders · 2024Article
- Longitudinal follow-up of metformin treatment in Fragile X Syndrome.Frontiers in psychology · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
To date, there has been limited research on the primary concerns and treatment priorities for individuals with fragile X syndrome (FXS) and their families. The National Fragile X Foundation in collaboration with clinical investigators from industry and academia constructed a survey to investigate the main symptoms, daily living challenges, family impact, and treatment priorities for individuals with FXS and their families, which was then distributed to a large mailing list. The survey included both structured questions focused on ranking difficulties as well as qualitative analysis of open-ended questions. It was completed by 467 participants, including 439 family members or caretakers (family members/caretakers) of someone with FXS, 20 professionals who work with a person with FXS, and 8 individuals with FXS. Respondents indicated three main general areas of concern: Anxiety, behavioral problems, and learning difficulties. Important differences were noted, based on the sex and age of the individual with FXS. The results highlight the top priorities for treatment development for family members/caretakers, as well as a small group of professionals, and an even smaller group of individuals with FXS, while demonstrating challenges with "voice of the patient" research in FXS.
Indexed as
Identifiers
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.