DM1 originates from complex genetic mutations that result in abnormalities of the muscle proteins, including the muscle-specific chloride channel ClC-1.

Working under the theory that abnormal inclusion of a region of the ClC-1 gene known as exon 7a in the ClC-1 mRNA (the intermediate between gene and protein) may play a role in the development of DM1, Charles Thornton and his colleagues at the University of Rochester, Rochester, discovered a powerful method of correcting this aberrant exon 7a inclusion and reversing myotonia in mouse models of DM1. A nucleic acid compound known as a morpholino antisense oligonucleotide was generated that would allow the muscle cells to skip over the erroneously included exon7a. When this compound was injected into mice with DM1-like disease, exclusion of the extra genetic segment in the ClC-1 mRNA restored the function of ClC-1 protein and eliminated myotonia in the mice. The authors therefore suggested that it might be possible to use this approach to treat myotonia in individuals with DM1.

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In addition, FDA has improved its Web page on advisory committees by providing better access to information about waivers granted for conflicts of interest. This Web page provides current information about upcoming advisory committee meetings and other updated information related to FDA's advisory committee processes. The Web site is at fda/oc/advisory/default.htm.

Finally, the FDA has recently posted the names of outside experts that it has named to a new risk communication advisory committee to make recommendations to FDA about how best to communicate the risks and benefits of FDA regulated products. More information about this advisory committee and the list of members can be found at fda/bbs/topics/NEWS/2007/NEW01739.html.

FDA's policies on advisory committees continue to be informed by new studies on conflicts of interest. The agency asked a consultant, Eastern Research Group, to study 16 recent advisory committees. The report highlights the difficulty of assembling highly qualified experts who are free of conflicts and finds that those who have received waivers appear to be significantly more qualified than those who have not received waivers. The full report is available online at fda/oc/advisory/ERGCOIreport.pdf.

So far this year the agency has convened 47 meetings of expert independent advisory committees to advise FDA on topics such as new gene therapies and the safety of children's cough and cold medicines.

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