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Neuromuscular

Clinical and statistical excellence for DMD, MG, ALS, SMA, and rare disease programs integrating functional and digital endpoints.

Clinical Data Excellence for Neuromuscular Disorders

Magnus CRO supports neuromuscular studies with expertise in disease-specific scales, digital biomarkers, longitudinal modeling, and regulatory documentation. Our approach balances sensitivity to disease progression with compliance and data integrity.

Endpoints and Functional Scales

  • Duchenne Muscular Dystrophy (DMD): 6-Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), timed tests, and loss-of-ambulation analysis.
  • Myasthenia Gravis (MG): MG-ADL, QMG, and MG Composite — correlation with pharmacodynamic and antibody levels.
  • Amyotrophic Lateral Sclerosis (ALS): ALSFRS-R and FVC slope modeling, time-to-tracheostomy and survival endpoints.
  • Integration of caregiver/PRO data and composite functional indices with MCID and responder thresholds.

Biomarker and Digital Data Integration

  • Wearable and sensor-based data capture (e.g., actigraphy, gait, upper-limb motion) linked with EDC datasets.
  • QC pipelines for continuous device data with signal validation and time-window harmonization.
  • Exploratory biomarker analysis (CK, dystrophin quantification, neurofilament light chain, genetic panels).
  • Central lab and imaging data reconciliation within CDISC SDTM and ADaM structures.

Statistical Modeling and Longitudinal Analysis

  • MMRM, mixed-effects, and spline-based models to capture progressive decline or stabilization trends.
  • Handling of missing data with MI, delta-adjusted, and tipping-point sensitivity analyses.
  • Customized estimand frameworks aligning with ICH E9(R1) for intercurrent event handling.
  • Time-to-loss-of-function and multi-domain responder analyses.

Deliverables and Regulatory Support

  • Study-specific Statistical Analysis Plans (SAPs), ADaM datasets, and TLFs aligned with disease-specific endpoints.
  • Comprehensive Data Management Plans (DMPs) and edit checks for complex longitudinal designs.
  • Integrated CSR narratives, reviewer guides, and regulatory responses (EMA, FDA, MHRA).

Partner with Magnus CRO for Neuromuscular Innovation

Collaborate with our neuromuscular experts to build robust clinical and statistical strategies that deliver regulatory-grade evidence in rare and complex indications.

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