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Zolgensma's long-term effects under the microscope in new 5-year study

NCT ID NCT05335876

Ongoing Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 2 times

Summary

This study follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective the treatment remains over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
onasemnogene abeparvovec (Zolgensma)
What this could lead to
If successful, this study could confirm that a one-time dose of Zolgensma provides lasting benefits and is safe over many years for people with spinal muscular atrophy.
What could go wrong
This is a follow-up study, not a new treatment test. It may reveal unknown long-term side effects, and results depend on how well patients did in earlier trials.

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.

Kennedy disease muscle tissue disorder muscular atrophy spinal muscular atrophy

As listed by the trial registrant

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

SPINAL MUSCULAR ATROPHY (SMA)

Contacts and locations

Locations

  • Child Hosp Of The Kings Daughters

    Norfolk, Virginia, 23507, United States

  • Novartis Investigative Site

    Sydney, New South Wales, 2031, Australia

  • Novartis Investigative Site

    Leuven, 3000, Belgium

  • Novartis Investigative Site

    Curitiba, Paraná, 81520-060, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 05403 000, Brazil

  • Novartis Investigative Site

    Montreal, Quebec, H4A 3J1, Canada

  • Novartis Investigative Site

    Chongqing, Chongqing Municipality, 400010, China

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510623, China

  • Novartis Investigative Site

    Chengdu, Sichuan, 610041, China

  • Novartis Investigative Site

    Beijing, 100034, China

  • Novartis Investigative Site

    Beijing, 100069, China

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Copenhagen, 2100 O, Denmark

  • Novartis Investigative Site

    Bron, 69677, France

  • Novartis Investigative Site

    Garches, 92380, France

  • Novartis Investigative Site

    Strasbourg, 67000, France

  • Novartis Investigative Site

    Toulouse, 31059, France

  • Novartis Investigative Site

    Roma, RM, 00168, Italy

  • Novartis Investigative Site

    Kurume, Fukuoka, 830-0011, Japan

  • Novartis Investigative Site

    Shinjuku Ku, Tokyo, 1628666, Japan

  • Novartis Investigative Site

    Kuala Lumpur, 50300, Malaysia

  • Novartis Investigative Site

    Kuala Lumpur, 59100, Malaysia

  • Novartis Investigative Site

    Utrecht, 3584 CX, Netherlands

  • Novartis Investigative Site

    Riyadh, 11211, Saudi Arabia

  • Novartis Investigative Site

    Singapore, 119074, Singapore

  • Novartis Investigative Site

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    Kaohsiung City, 80756, Taiwan

  • Novartis Investigative Site

    Taipei, 10002, Taiwan

  • Novartis Investigative Site

    Bangkok, 10700, Thailand

  • Novartis Investigative Site

    London, WC1N 3JH, United Kingdom

  • Novartis Investigative Site

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • Novartis Investigative Site

    Hanoi, 100000, Vietnam

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