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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHRU of Brest
NOT_YET_RECRUITINGBrest, 29609, France
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Department of Pediatrics - Neurology and Infectious Diseases Toulouse University Hospital - Children's Hospital
NOT_YET_RECRUITINGToulouse, 31059, France
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Hautepierre Hospital - Mother and Child Hospital
NOT_YET_RECRUITINGStrasbourg, 67200, France
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I-Motion Pediatric Clinical Trial Platform Armand Trousseau Hospital
NOT_YET_RECRUITINGParis, 75012, France
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Marseille University Hospital - Timone Hospital Department of Pediatric Neurology - Specialized Pediatrics and Child Medicine
NOT_YET_RECRUITINGMarseille, 13005, France
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Pediatric Neurology Swynghedauw Hospital
NOT_YET_RECRUITINGLille, 59037, France
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Pediatric Neurology and Resuscitation Raymond-Poincare Hospital
NOT_YET_RECRUITINGGarche, 92380, France
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Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital
RECRUITINGBron, Rhone, 69500, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can gene therapy help babies with SMA reach milestones? a Real-World review
- School transitions put to the test for children with a rare muscle disease
- Horseback therapy may boost movement and breathing in kids with Muscle-Weakening disease
- Newborn screening study aims to catch rare diseases at birth
- Gene therapy trial aims to halt Muscle-Wasting disease
- Higher dose of spinraza tested in SMA patients who already tried risdiplam