A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

PHASE2CompletedINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

December 21, 2018

Primary Completion Date

October 23, 2020

Study Completion Date

October 23, 2020

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

Ataluren

Ataluren will be administered as per the dose and schedule specified in the arm.

Trial Locations (9)

10032

Columbia University College of Physicians & Surgeons, New York

23507

Children's Hospital of the King's Daughters, Norfolk

55455

University of Minnesota, Minneapolis

66160

University of Kansas Medical Center, Kansas City

77030

Texas Children's Hospital, Houston

85016

Phoenix Childrens Hospital, Phoenix

90025

University of California, Los Angeles (UCLA), Los Angeles

95817

University of California (UC) Davis Medical Center, Sacramento

78229-3900

University of Texas Heath Science Center at San Antonio, San Antonio

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY