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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

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?
Print a page for my doctor

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%.

ClinicalTrials.gov NCT07125898