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.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for GENETIC DISEASES, INBORN are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre

    Saint Petersburg, 194100, Russia

  • All India Institute of Medical Sciences

    New Delhi, 110029, India

  • Apollo Children's Hospital Chennai

    Chennai, Tamil Nadu, 600006, India

  • Apollo Gleneagles Hospital

    Kolkata, West Bengal, 700054, India

  • Children's Hospital of Fudan University

    Shanghai, 200032, China

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Childrens Hospital London Health Sciences Centre

    London, Ontario, N6A5W9, Canada

  • Childrens Hospital of Eastern Ontario

    Ottawa, Ontario, K1H8L1, Canada

  • Christian Medical College Hospital Vellore

    Vellore, Tamil Nadu, 632004, India

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Columbia University College of Physicians & Surgeons

    New York, New York, 10032, United States

  • Cook Childrens Medical Center

    Fort Worth, Texas, 76104, United States

  • General Hospital of Chinese Armed Police Forces

    Beijing, 100039, China

  • Hospital Angeles Chihuahua

    Chihuahua City, 31217, Mexico

  • Hospital Tunku Azizah Kuala Lumpur

    Kuala Lumpur, 50586, Malaysia

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP

    São Paulo, 05403-000, Brazil

  • Hospital de Clínicas da Universidade Federal de Minas Gerais

    Belo Horizonte, 30130-100, Brazil

  • Indiana University Health - Riley Child Neurology

    Indianapolis, Indiana, 46202, United States

  • Instituto Nacional de Pediatría

    Mexico City, 04530, Mexico

  • Instituto Nacional de Rehabilitacion

    Tlalpan, 14389, Mexico

  • Istanbul University- Instanbul Medical Faculty

    Istanbul, 34093, Turkey (Türkiye)

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 80756, Taiwan

  • Loma Linda University Children's Hospital

    San Bernardino, California, 92408, United States

  • National Institute of Mental Health and Neurosciences

    Bengaluru, Karnataka, 560029, India

  • National Taiwan University Hospital

    Taipei, 10002, Taiwan

  • Nizam's Institute of Medical Sciences (NIMS)

    Hyderabad, Telangana, 500082, India

  • Northwest Florida Clinical Research Group, LLC

    Gulf Breeze, Florida, 32561, United States

  • P.D. Hinduja Hospital

    Māhīm, Maharashtra, 400016, India

  • PTC Clinical Site

    Multiple Locations, Japan

  • Panchshil Hospital

    Ahmedabad, Gujarat, 380005, India

  • Perth Children's Hospital

    Nedlands, Western Australia, 6009, Australia

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Postgraduate Institute of Medical Education and Research

    Chandigarh, 160012, India

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Queen Mary Hospital

    Hong Kong, SAR, Hong Kong

  • Queensland Children's Hospital

    South Brisbane, Q4101, Australia

  • 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

  • Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie

    Warsaw, 02-097, Poland

  • Samsung Medical Center

    Seoul, 06351, South Korea

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Seoul National University Hospital

    Seoul, 3080, South Korea

  • Shenzhen Children's Hospital

    Shenzhen, 518038, China

  • Shriners Hospital for Children

    Portland, Oregon, 97239, United States

  • Siriraj Hospital

    Bangkok, 10700, Thailand

  • Stanford University Medical Center

    Palo Alto, California, 94305, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • The Childrens Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • The First Affiliated Hospital of Fujian Medical University

    Fuzhou, 350005, China

  • The Royal Childrens Hospital

    Parkville, Victoria, 3052, Australia

  • UMHAT Sofiamed

    Sofia, 1793, Bulgaria

  • Universidade Federal do Rio de Janeiro

    Rio de Janeiro, 21.941-912, Brazil

  • University Malaya Medical Centre (UMMC)

    Pantai, 59100, Malaysia

  • University Of Utah

    Salt Lake City, Utah, 84112, United States

  • University of California (UC) Davis Medical Center

    Sacramento, California, 95817, United States

  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland

    Oakland, California, 94143, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Michigan - CS Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • University of Pittsburgh School of Medicine

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Puerto Rico - School of Medicine

    San Juan, 00936-5067, Puerto Rico

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78229-3900, United States

  • Xiangya Hospital Central South University

    Hunan, 410008, China

Conditions

Explore the condition pages connected to this study.