Biostrophin
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Biostrophin is a drug which may serve as a vehicle for gene therapy, in the treatment of Duchenne and Becker muscular dystrophy.[1]
As mutations in the gene which codes for the protein dystrophin is the underlying defect responsible for both disorders, biostrophin will deliver a genetically-engineered, functional copy of the gene at the molecular level to affected muscle cells.[1] Dosage, as well as a viable means for systemic release of the drug in patients, is currently being investigated with the use of both canine and primate animal models.[2]
Biostrophin is being manufactured by Asklepios BioPharmaceuticals, Inc., with funding provided by the Muscular Dystrophy Association.[3]
See also
Other drugs for Duchenne muscular dystrophy
- Ataluren
- Rimeporide (experimental)
References
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