Deflazacort
- Generic Name
- Deflazacort
- Brand Names
- Emflaza
- Drug Type
- Small Molecule
- Chemical Formula
- C25H31NO6
- CAS Number
- 14484-47-0
- Unique Ingredient Identifier
- KR5YZ6AE4B
- Background
Deflazacort, also known as Emflaza, is a corticosteroid prodrug used as an agent to manage Duchenne Muscular Dystrophy (DMD). It is marketed by Marathon Pharmaceuticals and was approved in February 2017 by the FDA.
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- Indication
⑴用于原发性肾上腺皮质功能减退、风湿病、胶原性疾病、皮肤病、变态反应性疾病、眼科疾病、暴发性和播散性肺结核、造血系统疾患、溃疡性结肠炎、特发性肾病综合征等。
⑵用于治疗杜氏肌营养不良（DMD）。
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
- First Posted Date
- 2012-05-23
- Last Posted Date
- 2022-08-12
- Lead Sponsor
- University of Rochester
- Target Recruit Count
- 196
- Registration Number
- NCT01603407
- Locations
- 🇮🇹
C. Besta Neurological Institute Foundation, Milan, Italy
🇨🇦Alberta Children's Hospital, Calgary, Alberta, Canada
🇬🇧Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom
Deflazacort in Dysferlinopathies
Phase 2
Completed
- Conditions
- Interventions
- First Posted Date
- 2007-09-10
- Last Posted Date
- 2015-09-02
- Lead Sponsor
- Ludwig-Maximilians - University of Munich
- Target Recruit Count
- 25
- Registration Number
- NCT00527228
- Locations
- 🇩🇪
Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany
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