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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
editOther drugs for Duchenne muscular dystrophy
edit- Ataluren
- Rimeporide (experimental)
References
edit- ^ a b Khurdayan VK, Bozzo J, Prous JR (Oct 2005). "Chronicles in drug discovery". Drug News & Perspectives. 18 (8): 517–522. doi:10.1358/dnp.2005.18.8.953409. ISSN 0214-0934. PMID 16391721.
- ^ http://www.ncbiotech.org/news_and_events/industry_news/duchenne.html Dec 2006 Archived October 17, 2007, at the Wayback Machine
- ^ "Asklepios Biopharmaceutical, Inc. Announces Cross License with GlaxoSmithKline". 2006-09-28.
External links
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