Key Takeaway
This long-running observational study underlies several FKRP publications. The registry says recruiting, but status and participation must be confirmed with the current study source.
Trial and Study Record
Clinical Trial Readiness for the Dystroglycanopathies
Prospective University of Iowa natural-history study collecting clinical, functional, respiratory, quality-of-life, and selected biospecimen data across dystroglycanopathies.
Observational studyNatural HistoryTrial ReadinessFamilies
Why this record matters
- The study is designed to describe early signs, symptoms, and natural history and to collect information useful for future trials.
- Published FKRP analyses on activity, illness-associated weakness, and ambulation prediction used participants or data from this study.
Eligibility and participation notes
- The registry describes evidence of a dystroglycanopathy through pathology, a documented variant in a known gene, or abnormal alpha-dystroglycan glycosylation in cultured fibroblasts.
- The record includes children and adults and describes a University of Iowa clinical evaluation with some measures repeated annually.
- Public criteria are not an eligibility decision, and the site listing does not prove that a particular person can participate now.
Limits and cautions
- The study began in 2006, criteria have changed over time, and the current record’s status was last verified by the study team in July 2025.
- Estimated enrollment and completion dates are registry projections, not confirmed participation capacity.
- The broader dystroglycanopathy population means not every procedure, result, or publication is FKRP-specific.
Primary Sources
Direct links for verification
Primary registry or publication source used for the structured study summary on this page.
Related Content
Related routes inside the site
Study Record
Global FKRP Registry
International FKRP patient-registry record designed to characterize the population, support standards of care work, and help identify participants for future studies. ClinicalTrials.gov currently lists the study status as Unknown because the record has not been verified since January 2024.
Study Record
Long-Term Development of Muscular Dystrophy Outcome Assessments
A 24-month observational study evaluating clinical and patient-reported outcome measures across selected muscular dystrophies, including LGMD subtypes.
Paper Summary
Predicting Loss of Ambulation in Limb Girdle Muscular Dystrophy R9
Longitudinal natural-history analysis relating timed motor tests to loss of functional ambulation in 97 people with LGMDR9.