Due to a measles case in the community, please call before coming to Seattle Children’s if you or your child has potential measles symptoms.

Masking and Visitation Changes: Due to high rates of respiratory illnesses in our community, we’ve made changes to our masking and visitation guidelines. Learn more.

Current Research Studies

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

DMD_AtalurenSFT

Phase: N/A
Clinicaltrials.gov ID: NCT01247207

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:

PI (Study Doctor): Seth Perlman
Study Team Member: Cisco Pascual, [email protected]

Seattle Children’s complies with applicable federal and other civil rights laws and does not discriminate, exclude people or treat them differently based on race, color, religion (creed), sex, gender identity or expression, sexual orientation, national origin (ancestry), age, disability, or any other status protected by applicable federal, state or local law. Financial assistance for medically necessary services is based on family income and hospital resources and is provided to children under age 21 whose primary residence is in Washington, Alaska, Montana or Idaho.