SPINRAZA study tracks movement in adults with spinal muscular atrophy
NCT ID NCT04159987
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINRAZA works in adults with this condition.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SPINRAZA (nusinersen)
What this could lead to
If successful, this study could confirm that SPINRAZA helps maintain or improve motor function in adults with SMA type II, supporting its continued use.
What could go wrong
This is a small, open-label study with only 20 participants and no placebo group, so results may not apply broadly. It tracks function but does not test a new treatment.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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APHP
Paris, Île-de-France Region, 94000, France
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CHRU de Lille
Lille, Hauts-de-France, 59000, France
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CHU de Lyon
Lyon, Auvergne-Rhône-Alpes, 69677, France
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CHU de Montpellier
Montpellier, Occitanie, 34090, France
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CHU de Nice
Nice, Provence-Alpes-Côte d'Azur Region, 06000, France
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CHU de Toulouse
Toulouse, Occitanie, 31059, France
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Hopital de la Timone - APHM
Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France