Development and Validation of a Disease‐Specific, Patient‐Reported Outcome Measure: The Myotonic Dystrophy Type 2 Health Index
Initial assessment of the MD2HI provides evidence that it is a valid and reliable PRO capable of quantifying a patient's perception of their disease burden to better detect clinically relevant changes in response to therapeutic intervention.
Authors
- Jennifer Weinstein (ORCID: https://orcid.org/0000-0002-3984-2120)
- Anika Varma (ORCID: https://orcid.org/0000-0001-9533-7113)
- Jamison Seabury (ORCID: https://orcid.org/0000-0002-8523-509X)
- Alicia Brocht
- John Heatwole
- Preshetha Kanagaiah (ORCID: https://orcid.org/0000-0002-4675-0910)
- Nuran Dilek
- Charlotte Engebrecht (ORCID: https://orcid.org/0009-0002-2982-8197)
- Chad Heatwole (ORCID: https://orcid.org/0000-0002-2900-6612)
- Christina Shupe
- Spencer Rosero (ORCID: https://orcid.org/0000-0003-1408-8325)
- Charlotte Irwin
Institutions
- Rochester Institute of Technology (US)
- Cornell University (US)
- Spencer Foundation (US)
- Unity Health System (US)
- University of Rochester (US)
Publication Details
- Journal
- Muscle & Nerve
- Published
- 2025-11-26
- DOI
- https://doi.org/10.1002/mus.70084
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Muscular Dystrophy Association
- University of Rochester