Actively Recruiting
Long-Term Study to Understand LAMA2-related Dystrophies in Children Aged 2 to 15 in France Tracking Muscle, Respiratory, Cognitive, and Quality of Life Changes Over 2 Years
Led by Institut de Myologie, France · Updated on 2024-12-12
40
Participants Needed
4
Research Sites
3 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying children with LAMA2-related dystrophies LAMA2-RD, a rare muscular condition, to better understand its natural course and characteristics. This observational study aims to create a well-defined group of patients in France for ongoing follow-up and to prepare for future clinical trials. The research focuses on multiple aspects of the disease including muscle function, breathing, cognitive abilities, quality of life, growth, and biomarkers.
CONDITIONS
Brief Title
Natural History Study of Children With LAMA2-related Dystrophies
Research Team
A
Andreea SEFERIAN, Dr
E
Erwan GASNIER, PhD
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