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Treatment with OnabotulinumtoxinA for Oromandibular Dystonia: A Systematic Review and Meta-Analysis.
Toxins (Basel)
December 2024
Department of Neurology, Tokushima University, Tokushima 770-8503, Japan.
Article Synopsis
- Oromandibular dystonia (OMD) is a condition causing muscle contractions in the jaw and related areas, and this study analyzed the effects of botulinum toxin (onabotulinumtoxinA) as a treatment.
- A meta-analysis of 26 studies with 1103 patients showed that 96.2% experienced a positive response to the injections, with 88.9% achieving significant improvement of over 50%.
- Although 17.8% of patients had adverse effects, mainly dysphagia (10.1%), the overall findings suggest that botulinum toxin is an effective treatment for OMD but further studies are needed for more conclusive results.
Dysphagia and Muscle Weakness Secondary to Botulinum Toxin Type A Treatment of Cervical Dystonia: A Drug Class Analysis of Prescribing Information.
Toxins (Basel)
October 2024
Revance Therapeutics, Nashville, TN 37203, USA.
Article Synopsis
- The primary treatment for cervical dystonia (CD) symptoms involves botulinum toxin type A (BoNTA) injections, but comparing the safety of different BoNTAs is challenging due to non-interchangeable activity units.
- A study examined the relationship between the incidence of dysphagia and muscle weakness—key adverse effects—across BoNTAs approved in the US, finding that adverse drug reaction rates correlate strongly with the core neurotoxin content.
- DaxibotulinumtoxinA (DAXI) showed a lower core neurotoxin amount compared to conventional BoNTAs, indicating a potentially better safety profile and fewer adverse effects for patients with CD.
Novel de novo heterozygous CACNA1A gene variant in generalised dystonia: a case report.
BMJ Neurol Open
June 2024
Molecular Pathology, Pathology and Clinical Laboratory Medicine Administration, KFMC, Riyadh, Saudi Arabia.
Background: Dystonia is a genetic or non-genetic movement disorder with typical patterned and twisting movements due to abnormal muscle contractions that may be associated with tremor. Genetic and phenotypic heterogeneity leads to variable clinical presentation.
Methodology: Next-generation sequencing technologies are being currently used in the workup of patients with inherited dystonia to determine the specific cause in the individuals with autosomal dominant, recessive, X-linked or mitochondrial inheritance patterns.
Efficacy and Safety of DaxibotulinumtoxinA for Injection in Cervical Dystonia: ASPEN-1 Phase 3 Randomized Controlled Trial.
Neurology
February 2024
From the Department of Neurosurgery and Neurological Sciences (C.L.C.), Rush University Medical Center, Chicago, IL; Parkinson's Disease Center and Movement Disorders Clinic (J.J.), Department of Neurology, Baylor College of Medicine, Houston, TX; Department of Neurology (R.A.H.), University of South Florida, Tampa, FL; Kansas City Bone & Joint Clinic (A.T.P.), Overland Park, KS; Department of Neurology (M.D.B.), Jagiellonian University, Krakow, Poland; Department of Neurology (E.E.), Regional Hospital Pardubice, Czech Republic; Revance Therapeutics, Inc (D.V., R.G.R., T.M.G.), Nashville, TN; and Blue Obsidian Consulting, LLC (R.G.R.), Redwood, CA.
Background And Objectives: ASPEN-1 was a phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, duration of response, and safety of 2 doses of DaxibotulinumtoxinA for Injection (DAXI), a novel botulinum toxin type A formulation in participants with cervical dystonia (CD).
Methods: Adults (aged 18-80 years) with moderate-to-severe CD (Toronto Western Spasmodic Torticollis Rating Scale [TWSTRS] total score ≥20) were enrolled at 60 sites across 9 countries in Europe and North America. Participants were randomized (3:3:1) to single-dose intramuscular DAXI 125U, 250U, or placebo and followed for up to 36 weeks after injection.
Expanding the Clinical Spectrum of -Related Neurodevelopmental Disorder.
Neurol Genet
December 2023
From the UO Genetica Medica (Andrea Pietra), IRCCS Azienda Ospedaliero-Universitaria di Bologna; Department of Medical and Surgical Sciences (Andrea Pietra, C.G.), Alma Mater Studiorum University of Bologna; IRCCS Istituto delle Scienze Neurologiche di Bologna (F.P.), Programma di Neurogenetica; IRCCS Istituto delle Scienze Neurologiche di Bologna (M.G., Antonella Pini, C.G.), UOC Neuropsichiatria dell'età Pediatrica; IRCCS Istituto delle Scienze Neurologiche di Bologna (M.M., C.F.), Programma di neuroradiologia con tecniche ad elevata complessità; Department of Biomedical and Neuromotor Sciences (R.C., G.C., V.C., D.M.C.), Alma Mater Studiorum University of Bologna; and UO Anatomia (G.C.), Istologia Patologica, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Italy.
Objectives: gene encodes for Upstream Binding Transcription Factor, an essential protein for RNA metabolism. A recurrent de novo variant (c.628G>A; p.
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