Objectives: To assess the prevalence of Kennedy's disease in the Vasa region of Western Finland.
Patients And Methods: Verification of diagnosis by molecular genetic techniques since 1995.
Results: Within 2 years we have been able to identify a large number of families with this disorder. We have arrived at a point prevalence of Kennedy's disease in the district of Vasa Central Hospital of 13 patients per 85,000 male inhabitants. Assuming an even distribution throughout the country, this would suggest hundreds of patients with this disorder in Finland.
Conclusion: Kennedy's disease is the most common motor neuron disorder in the Vasa region, exceeding the prevalence of ALS by a factor of two, and far more common than any of the other motor neuron disorders. The fact that none of our patients, despite previous examinations, had correct diagnoses before 1995, indicates that Kennedy's disease still might be relatively underdiagnosed.
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http://dx.doi.org/10.1111/j.1600-0404.1998.tb01732.x | DOI Listing |
Nucleic Acids Res
January 2025
Department of Paediatrics, University of Oxford, OX3 7TY Oxford, United Kingdom.
Nucleic acid nanostructures offer unique opportunities for biomedical applications due to their sequence-programmable structures and functions, which enable the design of complex responses to molecular cues. Control of the biological activity of therapeutic cargoes based on endogenous molecular signatures holds the potential to overcome major hurdles in translational research: cell specificity and off-target effects. Endogenous microRNAs (miRNAs) can be used to profile cell type and cell state, and are ideal inputs for RNA nanodevices.
View Article and Find Full Text PDFClin Exp Rheumatol
January 2025
UMass Chan Medical School and UMass Memorial Medical Center, Boston, MA, USA.
Objectives: To assess the efficacy and safety of an intra-articular (IA) CLK/DYRK inhibitor, lorecivivint (LOR), for the treatment of moderate to severe symptomatic knee osteoarthritis (OA).
Methods: This was a Phase 3, 28-week, multicentre, double-blind, placebo-controlled study evaluating the efficacy and safety of a single IA injection of LOR. Patients with ACR-defined knee OA, Kellgren-Lawrence (KL) grades 2-3, and pain Numeric Rating Scale (NRS) ≥4 and ≤8 in the target knee were randomised (1:1) to receive LOR 0.
J Am Vet Med Assoc
January 2025
2College of Veterinary Medicine, The Ohio State University, Columbus, OH.
Objective: To report ocular examination findings before and after total ear canal ablation and lateral bulla osteotomy (TECALBO) and ventral bulla osteotomy (VBO) in cats and dogs.
Methods: At The Ohio State University and MedVet Columbus, ophthalmic examinations were performed on client-owned animals for which a TECALBO or VBO was indicated. Examination findings and postoperative complications relating to ophthalmic and/or neuro-ophthalmic diseases were recorded prior to surgery (T0) and at 1 day (T1), 2 to 3 weeks (T14), and ± 2 months (T60) postoperatively.
Rheumatol Ther
February 2025
Biosplice Therapeutics, Inc., 9360 Towne Centre Dr, San Diego, CA, 92121, USA.
Introduction: Lorecivivint (LOR), a CDC-like kinase/dual-specificity tyrosine kinase (CLK/DYRK) inhibitor thought to modulate inflammatory and Wnt pathways, is being developed as a potential intra-articular knee osteoarthritis (OA) treatment. The objective of this trial was to evaluate long-term safety of LOR within an observational extension of two phase 2 trials.
Methods: This 60-month, observational extension study (NCT02951026) of a 12-month phase 2a trial (NCT02536833) and 6-month phase 2b trial (NCT03122860) was administratively closed after 36 months as data inferences became limited.
Stem Cell Res
February 2025
Department of Neurology, Research Institute of Neuromuscular and Neurodegenerative Diseases, Shandong Key Laboratory of Mitochondrial Medicine and Rare Diseases, Jinan, Shandong, China. Electronic address:
Abnormal trinucleotide CAG repeat expansions in exon 1 of the Androgen Receptor (AR) gene has been identified as the cause of Kennedy disease (KD). We generated and characterized a human induced pluripotent stem cell (iPSC) line from peripheral blood mononuclear cells (PBMC) of a patient with genetically confirmed KD. The pluripotency of these iPSCs was verified by the expression of several pluripotency markers at both RNA and protein levels, as well as their capability to differentiate into all three germ layers.
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