Merrilee Needham
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View article: Altered Transcriptome Signature in Primary Human Myotubes Exposed to Inclusion Body Myositis Serum: A Pilot Case Comparison of Anti-cN1A Positive and Negative Sera
Altered Transcriptome Signature in Primary Human Myotubes Exposed to Inclusion Body Myositis Serum: A Pilot Case Comparison of Anti-cN1A Positive and Negative Sera Open
Inclusion body myositis (IBM) is a late-onset, treatment-resistant inflammatory myopathy. Approximately half of IBM patients develop autoantibodies against cytosolic 5′-nucleotidase 1A (cN1A), but their role in disease pathogenesis remains…
View article: Altered Sensorimotor Neuroplasticity in the Subacute Period Following Burn Injury
Altered Sensorimotor Neuroplasticity in the Subacute Period Following Burn Injury Open
Purpose Burn injuries affect the central nervous system, and evidence suggests they induce changes in cortical excitability related to motor dysfunction. The current study investigated markers of neuroplasticity in the motor cortex and mot…
View article: Characterising Manual Dexterity, Motor Cortex Neuroplasticity and Intracortical Inhibition Long After Burn Injury
Characterising Manual Dexterity, Motor Cortex Neuroplasticity and Intracortical Inhibition Long After Burn Injury Open
Purpose Persistent motor dysfunction after burn injury may be due to altered motor cortex and corticospinal tract function. This study examined manual dexterity, motor cortex neuroplasticity, and intracortical inhibition in former burn pat…
View article: Item selection for the development and validation of a revised classification criteria for adult and juvenile idiopathic inflammatory myopathies: MyoROC project
Item selection for the development and validation of a revised classification criteria for adult and juvenile idiopathic inflammatory myopathies: MyoROC project Open
The new items that will be tested in the revised criteria were generated with input from a wide range of stakeholders and included, most importantly, MSA, pattern of weakness, skin changes, and additional diagnostic modalities. The next st…
View article: Investigating the effects of age and conditioning stimulation intensity on SMA–M1 connectivity in younger, middle-aged, and older adults
Investigating the effects of age and conditioning stimulation intensity on SMA–M1 connectivity in younger, middle-aged, and older adults Open
Purpose This study aimed to investigate bilateral motor control and connectivity between supplementary motor area (SMA) and primary motor cortex (M1) in younger, middle-aged, and older healthy adults. Methods 32 younger (mean age 22.8 ± 5.…
View article: Novel missense variants in <i>CFL2</i> affect F-actin depolymerisation and expand the disease spectrum of <i>CFL2</i>-related myopathy
Novel missense variants in <i>CFL2</i> affect F-actin depolymerisation and expand the disease spectrum of <i>CFL2</i>-related myopathy Open
Cofilin-2, encoded by CFL2, is an actin-binding protein essential for regulating actin filament dynamics in skeletal muscle. Biallelic variants in CFL2 are associated with an ultra-rare, early-onset myopathy typically presenting as nemalin…
View article: Expression and Site-Specific Biotinylation of Human Cytosolic 5′-Nucleotidase 1A in Escherichia coli
Expression and Site-Specific Biotinylation of Human Cytosolic 5′-Nucleotidase 1A in Escherichia coli Open
Autoantibodies targeting cytosolic 5′-nucleotidase 1A (cN1A) are found in several autoimmune diseases, including inclusion body myositis (IBM), Sjögren’s syndrome, and systemic lupus erythematosus. While they have diagnostic relevance for …
View article: POS1063 ULVIPRUBART PHARMACOKINETICS, PHARMACODYNAMICS (PK/PD), AND SAFETY: PHASE 1 STUDY RESULTS IN PATIENTS WITH INCLUSION BODY MYOSITIS (IBM)
POS1063 ULVIPRUBART PHARMACOKINETICS, PHARMACODYNAMICS (PK/PD), AND SAFETY: PHASE 1 STUDY RESULTS IN PATIENTS WITH INCLUSION BODY MYOSITIS (IBM) Open
View article: The incidence of anti-HMGCR immune-mediated necrotizing myopathy: an Australian and UK retrospective multi-site cohort study
The incidence of anti-HMGCR immune-mediated necrotizing myopathy: an Australian and UK retrospective multi-site cohort study Open
Objectives Immune-mediated necrotizing myopathy (IMNM) with autoantibodies targeting 3-hydroxy-3-methylglutaryl-CoA reductase (anti-HMGCR) is considered a rare complication of statin therapy. We calculate the incidence of anti-HMGCR IMNM a…
View article: Strapped for Strength: A Comparison Study of Dynamometry Techniques to Evaluate Knee Extensor Strength in Inclusion Body Myositis
Strapped for Strength: A Comparison Study of Dynamometry Techniques to Evaluate Knee Extensor Strength in Inclusion Body Myositis Open
Introduction/Aims Accurate measurement of knee‐extensor strength in people with inclusion body myositis (IBM) is vital to track disease progression and provide a standardized outcome for clinical trials. Isokinetic dynamometers are the cur…
View article: Reldesemtiv in Amyotrophic Lateral Sclerosis
Reldesemtiv in Amyotrophic Lateral Sclerosis Open
Importance Treatment options for amyotrophic lateral sclerosis (ALS) remain suboptimal. Results from a phase 2 study of reldesemtiv in ALS suggested that it may slow disease progression. Objective To assess the effect of reldesemtiv vs pla…
View article: Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis
Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis Open
Instrumental to the progress of the trial has been the collaboration between site teams, patient partnership and adaptability.
View article: Optimism in inclusion body myositis: a double-blind randomised controlled phase III trial investigating the effect of sirolimus on disease progression in patients with IBM as measured by the IBM Functional Rating Scale
Optimism in inclusion body myositis: a double-blind randomised controlled phase III trial investigating the effect of sirolimus on disease progression in patients with IBM as measured by the IBM Functional Rating Scale Open
This phase IIb/III trial builds on prior findings to assess sirolimus's potential in slowing or halting IBM progression, preserving patient function and independence, and advancing IBM therapeutic strategies and trial design.
View article: Mind over matter? A cross-sectional study exploring the influence of depression versus physical disability on patient-reported wellbeing in inclusion body myositis
Mind over matter? A cross-sectional study exploring the influence of depression versus physical disability on patient-reported wellbeing in inclusion body myositis Open
This cross-sectional study aimed to explore the relationships between physical disability, depression, and wellbeing in people with inclusion body myositis, a rare and progressively disabling muscle disease. The study investigated the rela…
View article: The relationship between patient-reported and clinician-assessed outcome measures in Inclusion body myositis - insights from a retrospective cohort study
The relationship between patient-reported and clinician-assessed outcome measures in Inclusion body myositis - insights from a retrospective cohort study Open
Inclusion body myositis (IBM) is an inflammatory myopathy, characterised by slow progression of weakness, skeletal muscle atrophy, and heterogeneous clinical presentation. This variability in disease progression and presentation complicate…
View article: Amyotrophic lateral sclerosis established as a multistep process across phenotypes
Amyotrophic lateral sclerosis established as a multistep process across phenotypes Open
Background and purpose Given the accepted multistep process of disease causation in amyotrophic lateral sclerosis (ALS), the present study was undertaken to determine the number of steps required for disease onset across each of the ALS ph…
View article: Correction: Testosterone treatment combined with exercise to improve muscle strength, physical function and quality of life in men affected by inclusion body myositis: A randomised, double-blind, placebo-controlled, crossover trial
Correction: Testosterone treatment combined with exercise to improve muscle strength, physical function and quality of life in men affected by inclusion body myositis: A randomised, double-blind, placebo-controlled, crossover trial Open
[This corrects the article DOI: 10.1371/journal.pone.0283394.].
View article: Prevalence of urinary and bowel incontinence among individuals with myositis: A cross-sectional study
Prevalence of urinary and bowel incontinence among individuals with myositis: A cross-sectional study Open
Background:: No evidenced link exists between myositis and incontinence, however myositis patients identify incontinence as an issue. This research was initiated in response to advocacy by consumers to investigate this topic. Objective:: T…
View article: Balancing Clinical Applicability and Scientific Depth in ML Models for MDA5-DM Prognosis
Balancing Clinical Applicability and Scientific Depth in ML Models for MDA5-DM Prognosis Open
International audience
View article: Response to: Correspondence on ‘Current myositis clinical trials and tribulations’ by Saygin et al
Response to: Correspondence on ‘Current myositis clinical trials and tribulations’ by Saygin et al Open
View article: Author Correction: International Guideline for Idiopathic Inflammatory Myopathy-Associated Cancer Screening: an International Myositis Assessment and Clinical Studies Group (IMACS) initiative
Author Correction: International Guideline for Idiopathic Inflammatory Myopathy-Associated Cancer Screening: an International Myositis Assessment and Clinical Studies Group (IMACS) initiative Open
View article: 272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16–18 June 2023, Hoofddorp, The Netherlands
272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16–18 June 2023, Hoofddorp, The Netherlands Open
Since the publication of the 2013 European Neuromuscular Center (ENMC) diagnostic criteria for Inclusion Body Myositis (IBM), several advances have been made regarding IBM epidemiology, pathogenesis, diagnostic tools, and clinical trial re…
View article: Does inspiratory muscle training improve lung function and quality of life in people with inclusion body myositis? A pilot study
Does inspiratory muscle training improve lung function and quality of life in people with inclusion body myositis? A pilot study Open
Inclusion body myositis is the most common acquired myositis in adults, predominantly weakening forearm flexor and knee extensor muscles. Subclinical respiratory muscle weakness has recently been recognised in people with inclusion body my…
View article: Clinical prognostic factors predicting survival of motor neuron disease patients with gastrostomy: A retrospective analysis
Clinical prognostic factors predicting survival of motor neuron disease patients with gastrostomy: A retrospective analysis Open
Introduction/Aims Enteral feeding via gastrostomy is a key intervention to prevent significant weight loss in Motor Neuron Disease (MND). The aim of this study was to explore demographic, clinical, and nutritional factors associated with s…
View article: Depression is a more significant predictor for wellbeing in Inclusion Body Myositis than physical disability
Depression is a more significant predictor for wellbeing in Inclusion Body Myositis than physical disability Open
Objectives (1) determine if there is a correlation between disability, depression, and wellbeing in people with IBM, (2) determine if disability and depression can predict wellbeing in people with IBM, and (3) identify the prevalence of de…
View article: Mind Over Matter? A Cross-Sectional Study Exploring the Influence of Depression Versus Physical Disability on Patient-Reported Wellbeing in Inclusion Body Myositis
Mind Over Matter? A Cross-Sectional Study Exploring the Influence of Depression Versus Physical Disability on Patient-Reported Wellbeing in Inclusion Body Myositis Open
View article: Identification of distinct immune signatures in inclusion body myositis by peripheral blood immunophenotyping using machine learning models
Identification of distinct immune signatures in inclusion body myositis by peripheral blood immunophenotyping using machine learning models Open
Objective Inclusion body myositis (IBM) is a progressive late‐onset muscle disease characterised by preferential weakness of quadriceps femoris and finger flexors, with elusive causes involving immune, degenerative, genetic and age‐related…
View article: Responsiveness and meaningful thresholds of PROMIS pain interference, fatigue, and physical function forms in adults with idiopathic inflammatory myopathies: Report from the OMERACT Myositis Working Group
Responsiveness and meaningful thresholds of PROMIS pain interference, fatigue, and physical function forms in adults with idiopathic inflammatory myopathies: Report from the OMERACT Myositis Working Group Open
View article: High-resolution HLA genotyping in inclusion body myositis refines 8.1 ancestral haplotype association to DRB1*03:01:01 and highlights pathogenic role of arginine-74 of DRβ1 chain
High-resolution HLA genotyping in inclusion body myositis refines 8.1 ancestral haplotype association to DRB1*03:01:01 and highlights pathogenic role of arginine-74 of DRβ1 chain Open
High-resolution HLA sequencing more precisely characterised the alleles associated with IBM and defined a haplotype linked to earlier disease onset. Identification of the critical amino acid residue by advanced biostatistical analysis of i…
View article: Clinical prognostic factors predicting survival of motor neuron disease patients with gastrostomy: a retrospective analysis
Clinical prognostic factors predicting survival of motor neuron disease patients with gastrostomy: a retrospective analysis Open