New hope for kids with SMA: experimental drug ARGX-119 enters phase 2 trial
NCT ID NCT07287982
First seen Dec 17, 2025 · Last updated May 24, 2026 · Updated 20 times
Summary
This study tests a new medicine, ARGX-119, in children aged 5 to 17 with spinal muscular atrophy (SMA). The goal is to find the right dose and see if it is safe and helps improve muscle strength and movement. Participants will receive either the study drug or a placebo for 24 weeks, then everyone can receive the active drug for up to 2 years. All children will continue their usual SMA treatments during the study.
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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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Contacts and locations
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Study contacts
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Contact
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Boston Children's Hospital
NOT_YET_RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital Philadelphia - Neurology
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Childrens Hospital of The Kings Daughters
NOT_YET_RECRUITINGNorfolk, Virginia, 23507, United States
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Columbia University Herbert Irving Comprehensive Cancer Center
NOT_YET_RECRUITINGNew York, New York, 10032, United States
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Connecticut Children's Medical Center
NOT_YET_RECRUITINGHartford, Connecticut, 06106, United States
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Neurology Rare Disease Center
RECRUITINGFlower Mound, Texas, 75028, United States
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Rady Childrens Hospital
NOT_YET_RECRUITINGSan Diego, California, 92123, United States
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Rare Disease Research FL LLC
RECRUITINGKissimmee, Florida, 34746, United States
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Rare Disease Research NC, LLC
RECRUITINGHillsborough, North Carolina, 27278, United States
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St. Jude Children's Research Hospital
NOT_YET_RECRUITINGMemphis, Tennessee, 38105, United States
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Stanford University Medical Center
NOT_YET_RECRUITINGStanford, California, 94305, United States
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Texas Children's Hospital
NOT_YET_RECRUITINGHouston, Texas, 77030, United States
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The Curators of the University of Missouri on behalf of University of Missouri Health Care
NOT_YET_RECRUITINGColumbia, Missouri, 65212, United States
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The Johns Hopkins Hospital
NOT_YET_RECRUITINGBaltimore, Maryland, 21205, United States
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University of Iowa Stead Family Children's Hospital
NOT_YET_RECRUITINGIowa City, Iowa, 52242, United States
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Conditions
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