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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]
![]() | This article has multiple issues. Please help
improve it or discuss these issues on the
talk page. (
Learn how and when to remove these template messages)
|
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]