Arbekacin as a therapeutic readthrough inducer for treatment of nonsense mutation-mediated Duchenne muscular dystrophy

Read the new article - November 5th 2011 about Arbekacin.

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Thank you Blazena. I had never heard about negamycin or Arbekacin. I knew of course about gentamycin, but ruled it out in my mind because of the well reported adverse effects. My son has a nonsense mutation and we have been waiting for nearly nine years to see how the PTC-124/Ataluren story finally unfolds (if ever). Maybe these Japanese researchers can come up with a substitute for Ataluren before the FDA makes up its mind on whether or not that 11-inch shortfall in the 6 minute walk test early last year really was a failure!. 

Anybody else knows about this?





Well, I have to reply to my own message, because I've just checked Arbekacin out and it turns out to be just another ultra-potent antibiotic, with the same toxicity issues that made gentamycin useless for this therapy.


This is from the Canadian website DrugBank: 


"Ototoxicity and nephrotoxicity are the most serious adverse effects of aminoglycoside therapy and are more likely to occur in patients with a history of renal impairment or who are receiving other ototoxic and/or nephrotoxic drugs. Normal duration of IM or IV aminoglycoside therapy is 7-10 days. Although a longer duration may be necessary in some cases, toxicity is more likely to occur when aminoglycoside treatment is continued for longer than 10 days"


I guess that those of us with boys suffering from a nonsense mutation will have to wait until the FDA stops fooling around with Ataluren....

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