Antisense oligonucleotide drug shows promising results

[Doug Carpenter]

This report :

https://mndresearch.blog/2019/05/07/tofersen-antisense-oligonucleotide-drug-shows-promising-results-in-phase-1-2-trial/

- on the MND research blog suggests promising phase 2 results specifically for SOD1-related MND.

Doug

[johnburton]

I went to the FathoM event in Oxford a few weeks ago. The lead researcher on the Sheffield SOD1 trial was there, with one of the trial participants. They conducted a conversation in front of the audience. In the course of it, the participant said that her symptoms had not progressed during the two years of the trial so far. The professionals of course made no such claim. After all, she could have been on the placebo. She had had around 19 monthly lumbar punctures so far, but said they didn't bother her.

The participant clearly had a slow moving form of MND, as she started with it 4 years before the trial started. She was in a manual wheelchair, but still had full use of her arms and hands and could speak normally and clearly.

I thought this sounded most promising.