SRP-6004
- Generic Name
- SRP-6004
- Brand Names
- -
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- -
- Unique Ingredient Identifier
- -
- Background
A recombinant AAVrh74 gene therapy expressing the dysferlin transgene under control of a muscle-specific promoter (rAAVrh74.MHCK7.DYSF.DV).
- Associated Conditions
- -
- Associated Therapies
- -
rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
- First Posted Date
- 2016-03-16
- Last Posted Date
- 2021-05-13
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 2
- Registration Number
- NCT02710500
- Locations
- 🇺🇸
Nationwide Children's Hosptial, Columbus, Ohio, United States
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