Research programme: micro dystrophin gene therapies - Institut de Myologie/Sarepta Therapeutics
Alternative Names: Micro Dystrophin gene therapyLatest Information Update: 28 Apr 2024
At a glance
- Originator Institut de Myologie; Sarepta Therapeutics
- Developer Sarepta Therapeutics
- Class Antisense oligonucleotides; Gene therapies; Morpholines
- Mechanism of Action Dystrophin expression stimulants; RNA interference; RNA splicing modulators
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- No development reported Duchenne muscular dystrophy
Most Recent Events
- 28 Apr 2024 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in France (Parenteral)
- 28 Apr 2024 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (Parenteral)