Key Takeaway
This study evaluates outcome measures across several muscular-dystrophy groups. It is not an FKRP treatment study, and the registry listing does not establish current site availability or individual eligibility.
Trial and 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.
Observational studyTrial ReadinessNatural HistoryFamilies
Why this record matters
- The primary focus is feasibility and suitability of standardized motor assessment, with additional functional, respiratory, health-index, and patient-reported measures.
- Blood samples may be retained for biomarker research; the registry states that no clinical diagnosis is provided through the study.
Eligibility and participation notes
- The record describes clinically affected, genetically confirmed participants aged 6 to 50 with an included LGMD, myotonic dystrophy type 2, or late-onset Pompe diagnosis.
- Selected criteria include protocol-defined proximal weakness, respiratory function, and no investigational-product trial participation.
- The study team must determine whether a specific LGMD subtype and individual meet the complete current protocol.
Limits and cautions
- The registry groups multiple diseases and does not identify an FKRP-specific enrollment or analysis in the public summary.
- Status was last verified by the responsible party in November 2025 and may not reflect current screening capacity.
- The estimated enrollment target is not a current actual count, and one listed site does not imply access for every reader.
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
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.
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.
Paper Summary
Childhood Activity on Progression in Limb Girdle Muscular Dystrophy 2I
Retrospective analysis relating self-reported middle-school activity to later motor and respiratory measures in 41 people with FKRP variants.