Goal of this study

To treat Duchenne muscular dystrophy.

 

Summary

Duchenne muscular dystrophy (DMD) is a genetic disease due to the absence of a protein called dystrophin. DMD is characterized by progressive muscle degeneration and weakness, and is due to deterioration of muscle cells which are important for body movement. This study will investigate the long-term efficacy of Ataluren in patients with DMD. Ataluren will be given to the patient through the mouth three times daily every day for a period of144 weeks.

 

Who can participate in this study?

Patients who have been diagnosed with Duchenne muscular dystrophy, genetically diagnosed with a nonsense mutation, and who are 5 years and older may be eligible to participate in this study. This study accepts boys and does not accept healthy volunteers.

Important: Additional eligibility criteria apply and the patient’s record will be verified by the study doctor (with the patient’s approval) to confirm eligibility.

 

ClinicalTrials.gov Identifier: NCT03179631

Contact Information

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