Current Research Studies

An Open ? Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy


What is the goal of the study?

This is an open label safety study for patients with DMD who have participated in previous clinical trials of ataluren. The purpose of this protocol is to provide continuing access to the investigational drug and to collect safety data for long term use.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team: