Podcast on C9 aso

[Nikki J]

After the huge biogen failure and their assertion that the whole approach was wrong we all wondered what would happen with the other 2 similar asos. WAVE was already in progress and has continued. The other c9 aso from Dr Brown and his lab was getting ready to start. They paused and Dr Brown said they would reassess after seeing Biogen’s data. Yesterday he and Dr Watts did a podcast indicating they are continuing to work on this and a trial is expected. Dr Brown said, as he has before, that he believes early intervention is key

Antisense Oligonucleotide Suppression of C9orf72

Dr. Jeff Ratliff discusses the suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide with Drs. Jonathan Watts and Robert Brown.

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[Haruba]

So sad that society does not recognize this as an ethical failure. Today they can essentially make c9 treatable and livable, yet they will not make available and will probably make people continue to die for at least five more years.

 

[Nikki J]

Do you mean the aso discussed in the podcast? A couple of points they proceeded as quickly as allowed by the fda who required more animal studies before anyone else could be dosed and they had to raise funds and obtain the animals which were in short supply and for the money they are academics not big pharma. There is a trial being prepared which will recruit early stage pals and a few later stage.

it is critical to realize that the man in this n=1 did not have full blown ALS. He is a c9 carrier who had some symptoms and emg changes.

yes it is very hopeful and I would take it in a heartbeat if offered to me but these doctors are doing the best they can. My own neurologist works closely with them and has updated me on this every time I see her.