Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Inclusion Criteria:

- Evidence of signed and dated informed consent/assent document(s) indicating that the
participant (and/or his parent/legal guardian) has been informed of all pertinent
aspects of the trial. Note: If the study candidate is considered a child under local
regulation, a parent or legal guardian must provide written consent prior to
initiation of study screening procedures and the study candidate may be required to
provide written assent. The rules of the responsible Institutional Review
Board/Independent Ethic Committee (IRB/IEC) regarding whether one or both parents must
provide consent and the appropriate ages for obtaining consent and assent from the
participant should be followed.

- History of exposure to ataluren in a prior PTC study or treatment plan in nmDBMD.

- In participants who are sexually active, willingness to abstain from sexual
intercourse or employ a barrier or medical method of contraception during ataluren
administration and the 6-week follow up period.

- Willingness and ability to comply with scheduled visits, drug administration plan,
study procedures, laboratory tests, and study restrictions Note: Psychological,
social, familial, or geographical factors that might preclude adequate study
participation should be considered.

Exclusion Criteria:

- Exposure to another investigational drug within 1 month prior to start of study
treatment.

- Eligibility for another ataluren clinical trial that is actively enrolling study
participants.

- Positive for Hepatitis B core antibody or Hepatitis C antibody at screening.

- Known hypersensitivity to any of the ingredients or excipients of ataluren (Litesse®
UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127
[poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla,
Cab-O-Sil® M5P [colloidal silica], magnesium stearate).

- Ongoing use of the systemic aminoglycoside therapy.

- Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or
laboratory abnormality that, in the investigator's opinion, could adversely affect the
safety of the participant or make it unlikely that follow-up would be completed.