Hope for slowing muscle loss in duchenne muscular dystrophy
NCT ID NCT03179631
Summary
This study tested whether a drug called ataluren could slow the progression of muscle weakness in boys with a specific genetic form of Duchenne muscular dystrophy. For 72 weeks, 360 participants received either ataluren or a placebo, followed by another 72 weeks where everyone received ataluren. The main goal was to see if the drug helped boys walk farther and maintain their ability to walk, climb stairs, and perform daily activities longer.
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Contacts and locations
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Locations
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"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
Saint Petersburg, 194100, Russia
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All India Institute of Medical Sciences
New Delhi, 110029, India
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Apollo Children's Hospital Chennai
Chennai, Tamil Nadu, 600006, India
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Apollo Gleneagles Hospital
Kolkata, West Bengal, 700054, India
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Children's Hospital of Fudan University
Shanghai, 200032, China
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Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
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Childrens Hospital London Health Sciences Centre
London, Ontario, N6A5W9, Canada
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Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1, Canada
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Christian Medical College Hospital Vellore
Vellore, Tamil Nadu, 632004, India
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Columbia University College of Physicians & Surgeons
New York, New York, 10032, United States
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Cook Childrens Medical Center
Fort Worth, Texas, 76104, United States
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General Hospital of Chinese Armed Police Forces
Beijing, 100039, China
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Hospital Angeles Chihuahua
Chihuahua City, 31217, Mexico
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Hospital Tunku Azizah Kuala Lumpur
Kuala Lumpur, 50586, Malaysia
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Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP
São Paulo, 05403-000, Brazil
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Hospital de Clínicas da Universidade Federal de Minas Gerais
Belo Horizonte, 30130-100, Brazil
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Indiana University Health - Riley Child Neurology
Indianapolis, Indiana, 46202, United States
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Instituto Nacional de Pediatría
Mexico City, 04530, Mexico
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Instituto Nacional de Rehabilitacion
Tlalpan, 14389, Mexico
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Istanbul University- Instanbul Medical Faculty
Istanbul, 34093, Turkey (Türkiye)
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 80756, Taiwan
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Loma Linda University Children's Hospital
San Bernardino, California, 92408, United States
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National Institute of Mental Health and Neurosciences
Bengaluru, Karnataka, 560029, India
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National Taiwan University Hospital
Taipei, 10002, Taiwan
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Nizam's Institute of Medical Sciences (NIMS)
Hyderabad, Telangana, 500082, India
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Northwest Florida Clinical Research Group, LLC
Gulf Breeze, Florida, 32561, United States
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P.D. Hinduja Hospital
Māhīm, Maharashtra, 400016, India
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PTC Clinical Site
Multiple Locations, Japan
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Panchshil Hospital
Ahmedabad, Gujarat, 380005, India
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Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
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Phoenix Childrens Hospital
Phoenix, Arizona, 85016, United States
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Postgraduate Institute of Medical Education and Research
Chandigarh, 160012, India
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Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, 50612, South Korea
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Queen Mary Hospital
Hong Kong, SAR, Hong Kong
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Queensland Children's Hospital
South Brisbane, Q4101, Australia
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Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
Moscow, 125412, Russia
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Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie
Warsaw, 02-097, Poland
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Samsung Medical Center
Seoul, 06351, South Korea
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Seoul National University Hospital
Seoul, 3080, South Korea
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Shenzhen Children's Hospital
Shenzhen, 518038, China
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Shriners Hospital for Children
Portland, Oregon, 97239, United States
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Siriraj Hospital
Bangkok, 10700, Thailand
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Stanford University Medical Center
Palo Alto, California, 94305, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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The Childrens Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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The First Affiliated Hospital of Fujian Medical University
Fuzhou, 350005, China
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The Royal Childrens Hospital
Parkville, Victoria, 3052, Australia
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UMHAT Sofiamed
Sofia, 1793, Bulgaria
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Universidade Federal do Rio de Janeiro
Rio de Janeiro, 21.941-912, Brazil
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University Malaya Medical Centre (UMMC)
Pantai, 59100, Malaysia
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University Of Utah
Salt Lake City, Utah, 84112, United States
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University of California (UC) Davis Medical Center
Sacramento, California, 95817, United States
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University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland
Oakland, California, 94143, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Michigan - CS Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15224, United States
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University of Puerto Rico - School of Medicine
San Juan, 00936-5067, Puerto Rico
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University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229-3900, United States
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Xiangya Hospital Central South University
Hunan, 410008, China
Conditions
Explore the condition pages connected to this study.