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Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
study id #: NCT02592941
condition: Duchenne Muscular Dystrophy
status: approved for marketingpurpose:
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.
mechanism of action: Glucocorticoid to delay decline in muscle strength
last updated: November 22, 2018
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.
• Confirmed diagnosis of Duchenne muscular dystrophy
• The patient is >=5 years old
• Current on all childhood vaccinations including the chicken pox vaccine
• History or current medication condition that could affect safety or poses an additional risk
• Hypersensitivity or allergic reaction to steroids or their formulations
PTC Therapeutics Receives FDA Approval for the Expansion of the EMFLAZA® (deflazacort) Labeling to Include Patients...PTC Therapeutics, Inc. today announced t...
Effect of Deflazacort and Prednisone on Muscle Enzymes in the Treatment of Duchenne Muscular DystrophyObjective: To describe the effects of d...
PTC Therapeutics Announces Publication of Data Demonstrating the Clinical Differentiated Benefit of DeflazacortPTC Therapeutics, Inc. today announced t...
Longitudinal Pulmonary Function Testing Outcome Measures in Duchenne Muscular Dystrophy: Long-term Natural History w...We describe changes in pulmonary functio...
Potential Mechanisms for Prolonged Loss of Ambulation with Deflazacort in Duchenne Muscular Dystrophy – Tolera...Objective: To summarize prolonged loss ...
The impact of deflazacort on puberty in Duchenne muscular dystrophyBackground: The routine use of glucocor...
ICER Document Reviews Effectiveness of 3 Duchenne MD TherapiesThe Institute for Clinical and Economic ...