|CI||Professor Kate Bushby|
|sites||Newcastle and London GOSH|
This is a phase III, open-label study of ataluren in nmDBMD (nonsense mutation Duchenne and Becker Muscular Dystrophies) patients. To be eligible patients would have taken part in a previous PTC-sponsored trial of ataluren at an investigator site.
Subjects will take ataluren for approximately 96 weeks, 3 times per day:
- morning, 10 mg/kg
- midday, 10 mg/kg
- and evening 20 mg/kg
Study assessments will be performed at clinic visits:
- during screening
- on the first day of ataluren dosing
- and then every 12 weeks during the ataluren treatment period
Primary Objective: to assess the long-term safety and tolerability of the stated doses.
Secondary objectives include the following:
- To determine the effect of ataluren on ambulation and other aspects of physical function in ambulatory patients*
- To assess the effect of ataluren on daily activities, upper limb function, and breathing in non-ambulatory patients**
- To assess patient and/or parent/caregiver reports of changes in disease status:
- Retrospectively during after participation in previous studies
- prospectively during the current study.
* able to run/walk 10 meters in less than 30 seconds
** unable to run/walk 10 meters in less than 30 seconds