A5014299636- Genetic Neurodegenerative Diseases
- Autism Spectrum Disorder Research
- Multiple Sclerosis Research Studies
- Family and Disability Support Research
- Peripheral Neuropathies and Disorders
- Child Nutrition and Feeding Issues
- Neurological disorders and treatments
- Pluripotent Stem Cells Research
- Stress Responses and Cortisol
- Nerve injury and regeneration
- Parkinson's Disease Mechanisms and Treatments
- Fibromyalgia and Chronic Fatigue Syndrome Research
- Botulinum Toxin and Related Neurological Disorders
- Immunodeficiency and Autoimmune Disorders
- Memory and Neural Mechanisms
- Epigenetics and DNA Methylation
- Tryptophan and brain disorders
- Neural and Behavioral Psychology Studies
- Polyomavirus and related diseases
- Research on Leishmaniasis Studies
- Infant Development and Preterm Care
- Health Systems, Economic Evaluations, Quality of Life
- SARS-CoV-2 and COVID-19 Research
- Fungal Plant Pathogen Control
- Genetics and Neurodevelopmental Disorders
Roche (United Kingdom)
2022-2025
The University of Sydney
2024
Palacký University Olomouc
2024
The University of Western Australia
2024
Düsseldorf University Hospital
2024
Inserm
2024
Heinrich Heine University Düsseldorf
2024
Université de Bordeaux
2024
Roche (Switzerland)
2021-2024
Cardiff University
2014-2023
To report the results of OCARINA II (NCT05232825), a Phase III, randomized, open-label, controlled study designed to demonstrate non-inferiority in serum exposure ocrelizumab (OCR) when administered via subcutaneous (SC) versus intravenous (IV) routes.
Background: Mobility assessment is essential for monitoring disease progression in people with multiple sclerosis (PwMS). Technologies such as wearable sensors show potential this purpose, but consensus needed to optimize collection and interpretation of digital measures PwMS. Objective: To propose a framework measuring interpreting key aspects impaired gait PwMS using smartphone worn at the waist level. Methods: The was developed on basis clinical understanding knowledge sensor signal...
Objectives: To develop within-patient meaningful score differences (MSDs) on the Bayley Scales of Infant Development, Fourth Edition (Bayley-4), and Vineland Adaptive Behavior Scales, Third (Vineland-3), for individuals with Angelman syndrome (AS). Methods: A Delphi method, involving a panel 19 caregivers AS, was used to establish MSDs Bayley-4 Vineland-3 Growth Scale Values. MSD defined as smallest change that would noticeably impact daily functioning an individual AS or family quality life...
Quantitative assessment of movement impairment in Huntington's disease (HD) is essential to monitoring progression. This paper aimed develop and validate a novel low cost, objective automated system for the evaluation upper limb HD order eliminate inconsistency assessor offer more sensitive, continuous scale. Patients with genetically confirmed healthy controls were recruited this observational study. Demographic data, including age (years), gender, unified rating scale total motor score...
Abstract Background Autism spectrum disorder (ASD) is a common and heterogeneous neurodevelopmental condition that characterized by the core symptoms of social communication difficulties restricted repetitive behaviors. At present, there an unmet medical need for therapies to ameliorate these in order improve quality life autistic individuals. However, several challenges are currently faced ASD community relating development pharmacotherapies, namely conduct clinical trials. Balovaptan V1a...
Huntington's disease (HD) is a progressive inherited neurodegenerative disorder, causing involuntary movement and cognitive problems, severely affecting the quality of life. Controlling upper limb function core feature daily activity can prove problematic for people with HD. The Money Box Test (MBT) has been developed purpose quantifying frequently seen in In this research, wearable highly sensitive accelerometers are used to collect acceleration hands chest during performance MBT. Using...
Objectives : Detection and prediction of disability progression is a significant unmet need in people with progressive multiple sclerosis (PwPMS). Government health agencies have deemed the use patient-reported outcomes measures (PROMs) clinical practice trials major strategic priority. Nevertheless, data documenting utility PROMs neurological diseases scarce. This study evaluates if assessment could track PwPMS. Methods EMerging blood BIOmarkers PROgressive Multiple Sclerosis (EmBioProMS)...
Huntington's disease (HD) is a debilitating, genetically inherited neurodegenerative disorder that results in early loss of medium spiny neurons from the striatum and subsequent degeneration cortical other subcortical brain regions. Behavioral changes manifest as range motor, cognitive, neuropsychiatric impairments. It has been established replacement degenerated with rat-derived fetal whole ganglionic eminence (rWGE) tissue can alleviate motor cognitive deficits preclinical rodent models...
Early treatment of multiple sclerosis (MS) reduces disease activity and the risk long-term progression. Effectiveness ocrelizumab is established in relapsing MS (RMS); however, data early RMS are lacking. We evaluated 4-year effectiveness safety as a first-line therapy treatment-naive patients with recently diagnosed relapsing-remitting (RRMS). ENSEMBLE was prospective, 4-year, international, multicenter, single-arm, open-label, phase IIIb study. Patients were naive, aged 18-55 years, had...
Angelman syndrome (AS) is a rare, heterogenous neurogenetic condition, which significantly impacts the lives of people with AS and their families. Valid reliable measures reporting key symptoms functional impairments are required to support development patient-centered therapies. We describe clinician- caregiver-reported, AS-specific Global Impression scales for incorporation into clinical trials. Best practice US Food Drug Administration guidance measure was followed input from expert...
The basal ganglia are implicated in a wide range of motor, cognitive and behavioral activities required for normal function. This region is predominantly affected Huntington's disease (HD), meaning that functional ability progressively worsens. However, outcome measures HD, particularly those the upper limb, limited there an imperative well-defined, quantitative measures. Here we describe development evaluation Moneybox test (MBT). novel, limb assessment was developed accordance with...
Abstract To better understand the impact of children’s autism spectrum disorder (ASD) severity on families, we evaluated pathways through which ASD affected child sleep quality, caregiver strain, and quality. In a cross-sectional analysis U.S.-wide Simons Foundation Powering Autism Research for Knowledge (SPARK) cohort. Participants were caregivers dependents with aged 3–17 years (N = 3150). We found that increased strongly affects strain Child quality was minor mediator increasing strain....
To explore, from the perspective of Study Partners (SPs; eg, caregivers) clinical trial participants with autism spectrum disorder (ASD), any changes experienced in socialization and communication over trial, how these manifested, impact had on autistic individual, SP, family. This helps interpret whether outcomes were meaningful.Interviews conducted SPs individuals ASD, without intellectual disability, 2 trials: 86 children (aged 5-12 years) or adolescents 13-17 who took part aV1ation...
The VinelandTM Adaptive Behavior Scale is often used in autism spectrum disorder (ASD) trials. Composite Score (VABS-ABC) the standardized overall score (the average of Socialization, Communication and Daily Living skills domains), 2-Domain (VABS-2DC) a novel outcome measure (average Socialization domains). A within-person meaningful change threshold (MCT) has not been established for VABS-2DC. This paper presents quantitative qualitative interpretation what constitutes these scores to...
To report results of the OCARINA I (NCT03972306) Phase Ib, dose-escalation study that assessed ocrelizumab (OCR) safety, tolerability and pharmacokinetic data in patients with relapsing primary progressive multiple sclerosis (RMS/PPMS), to select appropriate OCR subcutaneous (SC) dose for II.
Subcutaneous ocrelizumab is being developed to provide treatment flexibility and additional choice patients with multiple sclerosis. OCARINA I (NCT03972306) an open-label, multicenter, Phase 1b, dose-finding study investigate the pharmacokinetics, safety, tolerability, immunogenicity of subcutaneous select a dose for 3 II (NCT05232825).
<h3>Objective:</h3> To report changes over 4 years in patient-reported outcomes (PROs) people with relapsing-remitting multiple sclerosis (PwRRMS) who were switched to ocrelizumab after suboptimal response disease-modifying therapies (DMTs). <h3>Background:</h3> PwRRMS may experience increased symptom severity (particularly fatigue), impaired working capacity and compromised quality-of-life (QoL) despite treatment DMTs. The CASTING study (NCT02861014) examined PROs time ≥6 months of other...
The Stroop test is a widely used neuropsychological measuring attention and conflict resolution, which shows sensitivity across range of diseases, including Alzheimer's, Parkinson's Huntington's diseases. A rodent analogue the test, Response-Conflict task (rRCT), allows for systematic investigation neural systems underpinning performance in this test. Little known about involvement basal ganglia process. aim study was to use rRCT determine whether striatal subregions are recruited during...
Abstract Validated outcome measures with the capacity to reflect meaningful change are key assessing potential interventions for autism spectrum disorder (ASD). We derive clinically thresholds (MCTs) of Autism Impact Measure (AIM) and identify factors associated change. Baseline 12-months follow-up survey caregivers 2,761 children ASD aged 3–17 years from U.S. Simons Foundation Powering Research Knowledge (SPARK) cohort were analyzed. Using caregiver-reported anchors change, 12-month in...
Although rheumatoid arthritis (RA) is a disease of articular joints, patients often suffer from co-morbid neuropsychiatric changes, such as anxiety, that may reflect links between heightened systemic inflammation and abnormal regulation the hypothalamic-pituitary-adrenal (HPA) axis.
Abstract Background The Clinch Token Transfer Test (C3t) is a bi-manual coin transfer task that incorporates cognitive tasks to add complexity. This study explored the concurrent and convergent validity of C3t as simple, objective assessment impairment reflective disease severity in Huntington’s, not reliant on clinical expertise for administration. Methods One-hundred-and-five participants presenting with pre-manifest ( n = 16) or manifest (TFC-Stage-1 39; TFC-Stage-2 43; TFC-Stage-3 7)...
<h3>Introduction</h3> Cell transplantation is becoming a viable therapy for patients with Huntington’s disease (HD). Studies using rodent models of HD have identified need extended behavioural training to allow the recipient ‘learn use graft’. Thus, after intra-striatal grafting foetal tissue, striatally-dependent behaviours be re-established through targeted training. <h3>Aims</h3> To determine extent which cell can alleviate range cognitive deficits in HD, and identify optimal parameters...