Development of a signs and symptoms outcome measure for caregivers of patients with methylmalonic acidemia and propionic acidemia (MMAPAQ)
This study establishes the content validity of the MMAPAQ as the first ObsRO questionnaire for measuring core signs and symptoms of MMA and PA in clinical trials and community research. Scoring and psychometric measurement properties of the MMAPAQ will be established in future studies. The PedsQL™ was found to have face validity in measuring concepts that affect the MMA and PA patient populations and should also be considered for use in clinical trials in MMA and PA.
Authors
- Neha Durgam (ORCID: https://orcid.org/0000-0002-4371-7525)
- Geetanjoli Banerjee (ORCID: https://orcid.org/0000-0001-7023-5049)
- M. Alex Bellenger (ORCID: https://orcid.org/0000-0002-3564-2658)
- Ethan J. Schwartz
- Olga Moshkovich (ORCID: https://orcid.org/0000-0001-6464-8533)
- Vanja Sikirica (ORCID: https://orcid.org/0000-0003-1934-2222)
- Jerry E. Vockley (ORCID: https://orcid.org/0000-0002-8180-6457)
- Kathy Stagni
Institutions
- University of Pittsburgh (US)
- Minnesota Medical Association (US)
- UPMC Health System (US)
- Moderna Therapeutics (United States) (US)
- Imperial Consultants (GB)
- University of Pittsburgh Medical Center (US)
Publication Details
- Journal
- Molecular Genetics and Metabolism
- Published
- 2024-09-01
- DOI
- https://doi.org/10.1016/j.ymgme.2024.108577
- Citations
- 1
- Primary Topic
- Metabolism and Genetic Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.38
Funders
- Moderna