Can a spinal injection safely slow spinal muscular atrophy? a real-world study in korea seeks answers.
NCT ID NCT04317794
First seen Jul 27, 2026 · Last updated Jul 28, 2026 · Updated 1 time
Summary
This study tracks the safety and effectiveness of Spinraza (nusinersen) in people with spinal muscular atrophy (SMA) who receive the drug as part of routine care in Korea. Researchers will monitor for side effects and measure changes in motor skills over time. The goal is to see how well the treatment works outside of controlled clinical trials.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- nusinersen (Spinraza), a drug injected into the spinal fluid to treat spinal muscular atrophy
- What this could lead to
- If successful, this study could confirm that Spinraza is safe and effective for SMA patients in real-world Korean clinics.
- What could go wrong
- This is an observational study, not a controlled trial, so results may be less definitive. Side effects like injection-site reactions or serious adverse events are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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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Asan Medical Center
Seoul, South Korea
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Chonbuk National University Hospital
Jeonju, South Korea
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Chonnam National University Hospital
Gwangju, South Korea
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Chungbuk National University Hospital
Cheongju-si, South Korea
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Chungnam National University Hospital
Daejeon, South Korea
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Korea University Guro Hospital
Seoul, South Korea
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Korea university Anam Hospital
Seoul, South Korea
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Kyungpook National University Hospital
Daegu, South Korea
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Pusan Natioanl University Hospital
Busan, South Korea
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Pusan National University Yangsan Hospital
Yangsan, South Korea
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Samsung Changwon Hospital
Changwon, South Korea
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Samsung Medical Center
Seoul, South Korea
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Seoul Natioanl University Hospital
Seoul, South Korea
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Severance Hospital
Seoul, 03722, South Korea
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Severance Hospital, Yonsei University Health System
Seoul, South Korea
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Yeungnam University Hospital
Daegu, South Korea
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a nationwide registry unlock the secrets of adult spinal muscular atrophy?
- New drug BIIB115 aims to build on gene therapy for spinal muscular atrophy
- New hope for SMA babies: boosting gene therapy with a Follow-Up drug
- Real-World data reveals treatment patterns for kids with SMA
- Massive data dive reveals how SMA drugs perform outside the lab
- Higher doses of SMA drug tested for Long-Term safety