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  1. Home
  3. Multiple System Atrophy Combined Outcome Assessment (musyca): Process, Format, And Validation Plan.
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  3. Multiple System Atrophy Combined Outcome Assessment (musyca): Process, Format, And Validation Plan.

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Multiple System Atrophy Combined Outcome Assessment (MuSyCA): process, format, and validation plan.

Horacio Kaufmann1, Jose-Alberto Palma2, Patricio Millar Vernetti2

  • 1Department of Neurology, New York University Grossman School of Medicine, 530 First Av, Suite 9Q, New York, NY, 10016, USA. Horacio.kaufmann@nyulangone.org.

Clinical Autonomic Research : Official Journal of the Clinical Autonomic Research Society
|February 28, 2026

View abstract on PubMed

Summary
This summary is machine-generated.

A new tool, the Multiple System Atrophy Combined Outcome Assessment (MuSyCA), is developed for disease-modifying Multiple System Atrophy (MSA) clinical trials. MuSyCA enhances outcome assessment by combining multiple measures to track disease progression more effectively.

Keywords:
BiomarkersClinical outcome assessmentDisabilityNeurodegenerationProgression

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Area of Science:

  • Neuroscience
  • Clinical Trials
  • Biomarkers

Background:

  • The Unified Multiple System Atrophy Rating Scale (UMSARS) has limitations for clinical trial use in Multiple System Atrophy (MSA).
  • There is a need for comprehensive outcome measures to assess disease modification in MSA clinical trials.

Purpose of the Study:

  • To describe the development and validation plan for the Multiple System Atrophy Combined Outcome Assessment (MuSyCA).
  • To outline MuSyCA's structure, intended use, and assessment of its reliability, validity, and sensitivity in tracking MSA progression.

Main Methods:

  • Candidate outcome assessments identified via systematic literature review and longitudinal MSA cohort data analysis.
  • Content refined through Delphi-like consensus rounds with experts, patient advocates, and industry stakeholders.
  • Cognitive interviews with 20 MSA patients evaluated patient- and clinician-reported outcomes; validation includes construct validity, internal consistency, test-retest reliability, and responsiveness.

    • Main Results:

    • MuSyCA integrates patient- and clinician-reported outcomes, biomarkers (neurofilament light chain, neuroimaging), and performance-based measures.
    • This multimodal approach captures subjective and objective aspects of MSA progression, improving detection of treatment effects.
    • MuSyCA is specifically designed for clinical trials and not for clinical practice.

    Conclusions:

    • MuSyCA provides a multidimensional approach for precise, disease-relevant evaluations in trials for disease-modifying therapies in MSA.
    • Ongoing validation will establish MuSyCA as a standardized multimodal outcome measure, crucial for advancing MSA therapeutic development.