New study tracks how SMA kids change on modern treatments

NCT ID NCT06321965

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 60 children with spinal muscular atrophy (SMA) who are already receiving SMN-restoring therapies like nusinersen or gene therapy. Over two years, researchers will track their muscle function, fatigue, breathing, and thinking skills to describe new disease patterns that have emerged with these treatments. The goal is to better understand how SMA evolves today and to find better ways to monitor children in routine care and future trials.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • CHRU of Brest

    NOT_YET_RECRUITING

    Brest, 29609, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Department of Pediatrics - Neurology and Infectious Diseases Toulouse University Hospital - Children's Hospital

    NOT_YET_RECRUITING

    Toulouse, 31059, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Hautepierre Hospital - Mother and Child Hospital

    NOT_YET_RECRUITING

    Strasbourg, 67200, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • I-Motion Pediatric Clinical Trial Platform Armand Trousseau Hospital

    NOT_YET_RECRUITING

    Paris, 75012, France

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Marseille University Hospital - Timone Hospital Department of Pediatric Neurology - Specialized Pediatrics and Child Medicine

    NOT_YET_RECRUITING

    Marseille, 13005, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Pediatric Neurology Swynghedauw Hospital

    NOT_YET_RECRUITING

    Lille, 59037, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Pediatric Neurology and Resuscitation Raymond-Poincare Hospital

    NOT_YET_RECRUITING

    Garche, 92380, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital

    RECRUITING

    Bron, Rhone, 69500, France

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••