Charcot-Marie-Tooth UK

Pxt-3003 posible treatment for cmt1a

Hi! my name is Flory, i have 23 years old, i'm a girl from Rumania, and i have cmt1a.

Recently i read an article about the results of the trial with pleodrug phase 2, and i was very happy because in a e-mail that i receive from pharnext, they said that the trial may pass the next year in phase 3.There is the link where i read, and i wanted to to ask you what do you think about it?

Sory if i have gramatical errors.Thanks!

3 Replies

I was at a research conference this week, and the results were presented, and they do look promising, but there is still a very long way to go. The drug is made up of three different drugs combined and the next step will be to try to determine what is doing what.

But it does look promising. However, phase 3 trials are still a long way off because there are not yet adequate measures for type 1a that are sensitive to measure change properly. That issue has got to be addressed before the phase 3 trial can go ahead.



Thanks you very much for your answer! So isn't any chance to begin phase 3 the next year ?


I would like to ask you Where could we read the article about the results of the trial with pleodrug phase 2?


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