PAEDIATRIC NORTH STAR
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
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 |
| f._muntoni_et_al._categorizing_trajectories_and_individual_item_change_of_the_north_star_ambulatory_assessment_in_patients_with_duchenne_muscular_dystrophy.pdf |
| wms-2018-minimal-detectable-change-in-nsaa-final.pdf |
| bpna_poster_2020_brain_isoforms_and_motor_outcomes_in_dmd_final_poster_24jan2020.pdf |
| cardiorespiratory_progression_over_5_years_2020.pdf |
Historical Publications
| The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials |
| Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy |
| Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy |
| Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy |
| The NorthStar Ambulatory Assessment in Duchenne Muscular dystrophy: considerations for the design of clinical trials |
| Steroids in Duchenne muscular dystrophy |
| Development of a Functional Assessment Scale for Ambulatory Boys with Duchenne Muscular Dystrophy |
| The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials |
