No new drug, just monitoring: Long-Term safety check for DMD gene therapy recipients
NCT ID NCT05967351
First seen Feb 06, 2026 · Last updated May 15, 2026 · Updated 10 times
Summary
This study tracks 400 people with Duchenne muscular dystrophy who previously received the gene therapy SRP-9001. Researchers will monitor their health and muscle function over time to understand long-term safety and effectiveness. No new treatment is given in this study.
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This is a summary of
the original 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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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Chiildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Health Specialty Center
Dallas, Texas, 75207, United States
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Children's Hospital Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Children's Hospital of Colorado
Aurora, Colorado, 80045-7106, United States
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Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510, United States
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Columbia University Pediatric Neuromuscular Center
New York, New York, 10032, United States
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Great Ormond Street Hospital For Children NHS Foundation Trust
London, Greater London, WC1N 1EJ, United Kingdom
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Hong Kong Children's Hospital
Hong Kong, Hong Kong
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Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, 08950, Spain
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Hospital Universitario y Politecnico La fe. Neurology Department
Valencia, 46026, Spain
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Institute of Translational and Clinical Research
Newcastle upon Tyne, NE1 3BZ, United Kingdom
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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Kobe University Hospital
Kobe, Hyōgo, 650-0017, Japan
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Lenox Baker Children's Hospital (Duke University)
Durham, North Carolina, 27705, United States
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Lucile Packard Children's Hospital Stanford (LPCH)
Palo Alto, California, 94304, United States
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National Center of Neurology and Psychiatry
Kodaira, Tokyo, 187-8551, Japan
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National Taiwan University Hospital
Taipei, 100225, Taiwan
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Oregon Health and Science University
Portland, Oregon, 97239, United States
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Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, OX3 9DU, United Kingdom
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Rady Children's Hospital
San Diego, California, 92123, United States
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The Johns Hopkins Hospital, Rubenstein Child Health Bldg
Baltimore, Maryland, 21287, United States
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Tokyo Women's Medical University Hospital
Tokyo, 1628666, Japan
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico
Genova, 16147, Italy
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UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore
Roma, 00168, Italy
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Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)
Ghent, East Flanders, 9000, Belgium
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University of California, Davis
Sacramento, California, 95817, United States
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University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
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University of Iowa ICTS Clinical Research Unit
Iowa City, Iowa, 52242, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Utah Health
Salt Lake City, Utah, 84132, United States
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Universitätsklinikum Essen - Klinik für Kinderheilkunde I
Essen, North Rhine-Westphalia, 45147, Germany
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.