Study for young children with neonatal myotonic dystrophy type 1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study looks at measurements and child-focused outcomes in babies and toddlers with myotonic dystrophy type 1 (DM1) that started in the newborn period. It may help researchers better track how the condition affects children and evaluate care plans more accurately.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be from newborn up to 3 years and 11 months old when joining.
- The baby must have had DM1 symptoms within the first 30 days of life and needed hospital care (ward or NICU) for over 72 hours.
- Genetic testing must show an expanded CTG repeat in the DMPK gene (in the child or the mother).
- A parent or legal guardian must be able to consent and follow all study steps.
- Your child should not have another serious illness that could interfere with the study results.
- Your child should not have recent major trauma, a bleeding condition/very low platelets, metal inside the body (for scans), or a past allergic reaction to local numbing medicine.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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