Studying brain development in children with dystrophinopathies
Examines brain development in children with DMD to link gene changes with symptoms.
Plain-language title generated from the registry record
Registry title: Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
Key facts
- Where
- Paris, FranceSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 5 and 12 years old?
Source: NCT07125898, who can take part
Where in France
- Hopital Necker Enfants MaladesParis, FranceTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Assistance Publique - Hôpitaux de Paris
- Trial code
- NCT07125898
- Record updated
- 22 September 2026
- Original description
The study aims to identify a genotype/phenotype correlation by analyzing more finely the neurodevelopmental disorders in DMD patients.Read full original descriptionShow less
Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
The study aims to identify a genotype/phenotype correlation by analyzing more finely the neurodevelopmental disorders in DMD patients.
Inclusion: French citizenship, affiliated to the French Social Security,; 5 to 12 years old,; DMD diagnosis confirmed by a genetic analysis predicting breaking in the reading frame of the DMD gene with knowledge of the limits of the mutation,; Follow-up in a French referral or a skills center belonging to Filnemus.
Exclusion: Treatment with methylphenidate: In case of hyperactive patients, the treatment will be transitorily interrupted the week before testing; Cardiac dysfunction with left ventricular ejection fraction < 35%,; Severe intellectual deficiency with IQ < 55, and IQ < 70 for the deep neurocognitive evaluation (executive and socio-cognitive evaluation),; Cataract except if operated (pseudophakic),; High intraocular pressure,; Respiratory dysfunction with force vital capacity < 70%,; Difficulties in fine motor skills with D3 MFM scale < 75%.