ReviewJournal of molecular neuroscience : MN2016
Clinical Trials in Spinal and Bulbar Muscular Atrophy-Past, Present, and Future.
Review in Journal of molecular neuroscience : MN, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07443449 (Connecting SBMA National Registries/Databases), which is not on this map. Cited by 14 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.
Connecting SBMA National Registries/Databases: a Retrospective Study
Who cites it
14 citing papers in PubMed.
- Progressive Proximal Muscle Weakness Due to a 51 CAG Repeat Expansion in Exon 1 of the Androgen Receptor Gene: A Case Report of Kennedy Disease.The American journal of case reports · 2026Article
- Dynamic Balance in Spinal and Bulbar Muscular Atrophy: Relationship between Strength and Performance of Forward Lunge, Step Up and Over, and Step Quick Turn.Rehabilitation research and practice · 2021Article
- The French national protocol for Kennedy's disease (SBMA): consensus diagnostic and management recommendations.Orphanet journal of rare diseases · 2020Article
- Preliminary design and validation of the "6-K-scale" for bulbar symptoms evaluation in SBMA.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2019Article
- Disease AffectsInternational journal of molecular sciences · 2019Article
- Kennedy's disease (spinal and bulbar muscular atrophy): a clinically oriented review of a rare disease.Journal of neurology · 2019Review
- The metabolic and endocrine characteristics in spinal and bulbar muscular atrophy.Journal of neurology · 2018Article
- Biomarkers of Spinal and Bulbar Muscle Atrophy (SBMA): A Comprehensive Review.Frontiers in neurology · 2018Review
- Identification of neuron selective androgen receptor inhibitors.World journal of biological chemistry · 2017Article
- Article
- Previously Undiagnosed Spinal and Bulbar Muscular Atrophy as a Cause of Airway Obstruction after Robot-Assisted Laparoscopic Prostatectomy.Case reports in anesthesiology · 2017Article
- Validation of the Italian version of the SBMA Functional Rating Scale as outcome measure.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2016Article
- Introduction to the Special Issue on Spinal and Bulbar Muscular Atrophy.Journal of molecular neuroscience : MN · 2016Article
- Towards a European Registry and Biorepository for Patients with Spinal and Bulbar Muscular Atrophy.Journal of molecular neuroscience : MN · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
Abstract
Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy's disease, is a rare adult-onset lower motor neuron disorder with a classic X-linked inheritance pattern. It is caused by the abnormal expansion of the CAG-repeat tract in the androgen receptor gene. Despite important progress in the understanding of the molecular pathogenesis and the availability of a broad set of model organisms, successful translation of these insights into clinical interventions remains elusive. Here we review the available information on clinical trials in SBMA and discuss the challenges and pitfalls that impede therapy development. Two important factors are the variability of the complex neuro-endocrinological phenotype and the comparatively low incidence of the disease that renders recruitment for clinical trials demanding. We propose that these challenges can be and need to be overcome by fostering closer collaborations between clinical research centers, the patient communities and the industry and non-industry sponsors of clinical trials.
Indexed as
Identifiers
26572537What 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.