Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
PTC Therapeutics
With
Parexel
Dohmen Life Science Services
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers, as appropriate. Treating physicians will continue to follow their standard of care activities and procedures for management of DMD. Treating physicians participating in the expanded access program are required to collect/document any patient or caregiver reported safety events and report to the sponsor..
Age
5–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Confirmed diagnosis of Duchenne muscular dystrophy
The patient is ≥ 5 years old
Current on all childhood vaccinations including the chicken pox vaccine
You may not be if
History or current medication condition that could affect safety or poses an additional risk
Hypersensitivity or allergic reaction to steroids or their formulations
