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PAEDIATRIC NORTH STAR

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Clinical Trials.gov
MDC Clinical Trials Webpage

Current Approved Audits

  • Functional outcomes and trajectory, and the influence of factors affecting functional trajectory in younger children (3-5 years) with DMD. -Collaborators: North Star Clinical Network.
  • Longitudinal growth patterns in DMD patients from the first years of life to adolescence and factors affecting growth patterns and implications on nutritional management of DMD children. - Collaborators: North Star Clinical Network.
  • Exploration of DMD patient trajectory, particularly in relation to change in NSAA over a one year time period, and predicting functional trajectories in DMD children. - Collaborators: North Star Clinical Network and Collaborative Trajectory Analysis Project (cTAP).
  • Ataluren Managed Access Agreement - Collaborators: North Star Clinical Network, PTC Therapeutics, NHS England, National Institute for Health and Care Excellence (NICE), Muscular Dystrophy UK (MDUK), Action Duchenne (AD). 
  • Comparison of functional progression of disease in patients with Duchenne Muscular Dystrophy (DMD) treated with SRP-4053, an exon skipping therapy, with the natural history of patients with DMD from the NorthStar UK clinical registry. Collaborators: North Star Clinical Network and Collaborative Trajectory Analysis Project (cTAP).
  • Development of predictive machine learning algorithms using NorthStar Ambulatory Assessment data in DMD. Collaborators: North Star Network, Certus Technology Associated Ltd and Environmental Futures & Big Data Impact Lab, Exeter.
  • Evaluation of longitudinal trajectory of ambulatory function in a natural history cohort to support the clinical trial study design for a first in man multicentric clinical trial of AAV mediated microdystrophin gene therapy in DMD patients. Collaborators: North Star Network and Genethon, France.
Historical Audits
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Current Publications

s_joseph_et_al._fractures_and_linear_growth_in_a_nationwide_cohort_of_boys_with_duchenne_muscular_dystrophy_with_and_without_glucocorticoid_treatment.pdf
File Size: 371 kb
File Type: pdf
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f._muntoni_et_al._categorizing_trajectories_and_individual_item_change_of_the_north_star_ambulatory_assessment_in_patients_with_duchenne_muscular_dystrophy.pdf
File Size: 2074 kb
File Type: pdf
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wms-2018-minimal-detectable-change-in-nsaa-final.pdf
File Size: 43 kb
File Type: pdf
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bpna_poster_2020_brain_isoforms_and_motor_outcomes_in_dmd_final_poster_24jan2020.pdf
File Size: 761 kb
File Type: pdf
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cardiorespiratory_progression_over_5_years_2020.pdf
File Size: 603 kb
File Type: pdf
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Historical Publications
The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
File Size: 995 kb
File Type: pdf
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Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy
File Size: 390 kb
File Type: pdf
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Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy
File Size: 382 kb
File Type: pdf
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Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
File Size: 339 kb
File Type: pdf
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The NorthStar Ambulatory Assessment in Duchenne Muscular dystrophy: considerations for the design of clinical trials
File Size: 967 kb
File Type: pdf
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Steroids in Duchenne muscular dystrophy
File Size: 219 kb
File Type: pdf
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Development of a Functional Assessment Scale for Ambulatory Boys with Duchenne Muscular Dystrophy
File Size: 144 kb
File Type: pdf
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The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
File Size: 559 kb
File Type: pdf
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  • Home
  • NETWORK CENTRES
  • NETWORK GROUPS
  • NEWS AND EVENTS
  • CURRENT APPROVED AUDITS AND RESEARCH
  • CLINICAL TRIALS
  • GDPR
  • Contact us