Study Summary
The MSP study will prospectively and systematically study patients with multisystem proteinopathy. All enrolled participants will undergo three in-person and two remote visits, with the option to continue with annual in-person visits. All visits will include motor and functional assessments, and in-person visits will also include neurological and cognitive assessments along with collection of biological fluids.
For Diseases
- Multisystem proteinpathy (MSP)
Background
The goals of the study are to better understand how MSP changes people’s strength and movements as well as how it affects their daily life over time. Another goal is to collect biological specimens for future biomarker discovery. All of this will help researchers better plan and design future research studies.
Enrollment Criteria
- Genetically confirmed mutation in gene likely to cause MSP e.g. a variant in VCP (i.e. MSP1, also known as IBMPFD: Inclusion Body Myopathy, Paget’s Disease of Bone, and Frontotemporal Dementia), HNRNPA1, HNRNPA2B1, SQSTM1, or MATR3 gene
- Age 18 years or older at time of enrollment
- Able and willing to comply with study procedures
How to Participate:
In order to participate in this study, please contact the study coordinator at one of the participating institutions by phone or by e-mail, or contact the CReATe Consortium at projectcreate@med.miami.edu.
Travel support for this study provided by Cure VCP Disease.
