U.S., Aug. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07125040) titled 'Characterization of the Natural History of LAMA2-RD and Identification of Novel Disease Biomarkers' on July 17.
Brief Summary: The goal of this observational study is to learn about the natural history and multi-organ involvement of Laminin-Alpha-2-Related Dystrophy (LAMA2-RD) in pediatric and adult patients. The main questions it aims to answer are:
* What is the prevalence and nature of cardiac involvement, and how do this relate to age and muscular phenotype?
* What is the prevalence of peripheral neuropathy, and how do this relate to age and muscular phenotype?
* What is the extent of respiratory, nutritional, skeletal, and c...