Now that both companies have released results, does it seem like their drugs are comparable?  I find it hard to tell, but it seems like AviBiopharma is getting better results (although at a higher dose than GSK) and is reporting no drug related side effects.  GSK, it would seem is only using a low dose in their phase 3 trial and there seems to be some kidney problems that are caused by their drug.  Does anyone have an opinion?  My son has a deletion of 49 and 50 and we are considering trying to get him enrolled in GSK's clinical trial.  It is so hard to know what to do.

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Jennifer, am also in same dilemma. Am in India and am planning to relocate to Canada in hope of some solution and improvement. My son's characteristics are just similar to your son and i believe their age group is also same, My son is 5.5 yrs old. Main problem is that AVI is not accepting more patients for trial whereas GSK is. In case AVI doesn't come up with their drug in market in next few months then kids will suffer more and we will have no options at that time. Complicated disease and difficult decisons. Need suggestions and guidance.

i guess you have to decide if you want the alternative of not having particpated,and holding off what potentially could stall your sons deterioration.If my son was suitable i would try it and i am sure they will monitor all adverse events.It is a very personal choice...as parents who want to do the best for our sons we do not have many great alternatives.Only my thoughts and i know it is not easy of putting your son into trials.All trials carry risk.

It is difficult to say since the AVI trial was dose ranging and only one boy in the 20 mg/kg dose group showed significant (55%) dystrophin positive fibers. In the GSK 24-week extension trial they reported 60-100% positive fibers. More importantly GSK noticed variable improvement in the 6-min walk test with some boys improving while others DID NOT. AVI did not see any improvement.To date, they do not have a clear understanding if there is a correlation b/w % dystrophin and functional improvement, which is actually what would get a drug approved (not the dystrophin, but the functional improvement)!

 

As for the timelines, AVI will start a very small 24-week trial at higher doses, boys are already selected/waiting for this one. Then next phase, larger pivotal trial will start end of 2012 the earliest. GSK will start a phase 2, 48-week trial this Fall, however I think they plan to to have 3 groups: 3 mg/kg (LOWER than what they use in Europe and other countries around the World) as well as 6 mg/kg and placebo.

 

Difficult decision!

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