New gene therapy aims to slow duchenne muscular dystrophy
NCT ID NCT05881408
First seen May 22, 2026 · Last updated May 22, 2026
Summary
This study tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy, a severe muscle-weakening disease. About 148 males, both those who can still walk and those who cannot, will receive either the therapy or a placebo. The goal is to see if the treatment improves arm and lung function over 72 weeks.
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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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Centre Hospitalier Universitaire de Québec - Université Laval (pavillon Centre Hospitalier Universitaire Laval)
Québec, G1V 4G2, Canada
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510, United States
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Gangnam Severance Hospital, Yonsei University Health System
Seoul, 06273, South Korea
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Great Ormond Street Hospital for Children Foundation Trust
London, Greater London, WC1N 3JH, 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 Universitari Politecnic La Fe
Valencia, 46026, Spain
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IRCCS Istituto Neurologico Carlo Besta Neurepsichiatria Infantile 2 - Epilettologia e Neurologia dello Sviluppo
Milan, 20133, Italy
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Institute of Neruology, Schneider Children's Medical Center of Israel
Petah Tikva, Israel
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Institute of Translational and Clinical Research
Newcastle upon Tyne, NE13BZ, United Kingdom
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan
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Karolinska Universitetssjukhuset/Astrid Lindgrens Barnsjukhus, Barnneurologen
Solna, 171 76, Sweden
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Kyungpook National University Hospital
Daegu, 41944, South Korea
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LMU- Klinikum der Universitat Munchen, Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Abeteilung Neuropadiatrie, Campus Innenstadt
München, Bavaria, Germany
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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
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 Hospital Organization Osaka Toneyama Medical Center
Toyonaka-shi, Osaka, 560-8552, Japan
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National Taiwan University Hospital
Taipei, Taiwan
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Oxford University Hospitals NHS Foundation Trust
Oxford, Oxfordshire, United Kingdom
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Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, 50612, South Korea
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Rady Children's Hospital-San Diego
San Diego, California, 92123, United States
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Research Institute McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
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Sahlgrenska Universitetssjukhuset
Gothenburg, 416 85, Sweden
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Seoul National University Hospital
Seoul, NAP, 03080, South Korea
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Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
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The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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The Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1, Canada
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The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, Maryland, 21287, United States
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The Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo, 162-866, Japan
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U.O.S.D Centro Traslazionale di Miologia e Patologie Neurodegenerative, 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
Roma, 00168, Italy
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Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, 9000, Belgium
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Universitatsklinikum Essen, Klinik fur Kinderheilkunde I, Abteilung Neuropadiatrie Essen
Essen, North Rhine-Westphalia, Germany
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Universitatsklinikum Hamburg Eppendorf
Hamburg, Germany
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University of California at Davis Medical Center
Sacramento, California, 95817, United States
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University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, 32608, United States
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University of Rochester, Department of Neurology
Rochester, New York, 14642, United States
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Washington University of St. Louis, St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Conditions
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