The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Validate the change over 24 months using the Neuromuscular Gross Motor Outcome
Timeframe: 24 months
Validate the change over 24 months using the Bayley Scales of Infant and Toddler Development™ 4th Ed (BayleyTM-4)
Timeframe: 24 months
Validate the change over 24 months using the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders
Timeframe: 24 months
Validate the change over 24 months using the Motor Function Measure Scale-Short Form
Timeframe: 24 months
Validate the change over 24 months using the World Health Organization motor milestones
Timeframe: 24 months