Good news for those like me that need 52 skipping! Prosensa has published its next skipping plan. They are now in 50% of preclinical for skipping exons 45, 52 and 53. We are just praying...


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That seems to be pretty exciting,even I am waiting for some good news.Fingers crossed ................
where did they publish it? unable to find updated infor on US trials on their website?
Look here jenn

jenn said:
where did they publish it? unable to find updated infor on US trials on their website?
What's the latest on getting subsequent formulations approved? Once the first skip is proven to be effective and safe, will the others be rapidly approved or will they need to go through the same process?

Based on AVI BioPharma's website (, my son needs 53 skipped (he's missing 46-52) and I'm wondering if it'll be fast-tracked once 51's approved...

I wish Prosensa and AVI BioPharma would work on different exons. Seems like they're both working on the same exon (51)...

Praise the Lord! Has anyone heard of when this will happen. My son, has a deletion of 51 which means he needs a 52 skip....right?
It looks that first product approval will help to speed up the next ones. Prosensa is using the same type of molecules but AVI has changed from PMO technology in 51 skipping to PPMO in 50. (, that will make some differences in structure of molecules even when they are using the same mechanism. I know when they are similar in structure, then it will be more easy to get approval for the next.
If there is any possibility for shortening different phases (for example to combine safety, efficacy and dose scaling trials all together in one phase), this will makes the drug to be delivered several years sooner. I am ready to accept all risks that is toward my Daniel and there are many families like us.
There is not any color darker than black, we are already there.


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