Nonaminoglycoside compounds induce readthrough of nonsense mutations

Two compounds demonstrated readthrough activity in mdx mouse myotube cells carrying a nonsense mutation and induced significant amounts of dystrophin protein.

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Hi Ofir,

Sadly, this compound made by PTC was not able to improve things much for DMD patients. The article is dated 2009 and PTC started, then stopped, a Clinical Trial where they discovered how this medicine reacted in man vs mouse. Sure glad science has cured DMD in mice. Wish something would work for our children.

God Bless
The compound made by PTC (PTC 124 or Ataluren) was selected using an assay that can be wrong. But this two compounds were selected using a new assay that is better. I still believe that Ataluren can arrive to the market because the low dose did show some improvement. They had to stop the trial because the high dose didnt work. But this two compounds may work better then ataluren.
I have read the article by Auld and the response by Goetz and the follow ups, about the way they discovered the PTC 124. It is true that the assay used to discover PTC 124 could have been flawed to an extent yet at the same time, it still has some form of nonsense mutation read through ability.
It would be interesting to see what became of these 2 molecules they discovered and whether anything further was done with them. These might be the molecules that PTC refered to when they said ages ago that if the Ataluren didnt work they had other molecules they could work with

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