IRSF Scientific Meeting, Daybue and More Posted on June 19, 2023...

IRSF Scientific Meeting, Daybue and More
Posted on June 19, 2023 by melelllan

On June 5-7 IRSF held its Scientific Meeting. You’ve got to check out this line up! Acadia, Taysha, Neurogene, and another Australian company called NeuroTec all presented. Anavex did not present as this meeting involves new discoveries and information and they had no new information to share since the last meeting. I have to say on one hand I’m super excited about the number and diversity of presentation; on the other hand, I don’t understand a lot of the subject matter, lol.


On June 5, Dr. Ponni Subbiah, Acadia Pharmaceuticals presented LILAC data and introduced a new Real World Efficacy study, called LOTUS, to collect clinical data from Daybue families when they start commercial supply. There is no cost to families, and Acadia hopes caregivers will consider participating. I hope so, too; this is an incredible opportunity for us as a community to help physicians, researchers, Acadia, Neuren and other families down the road.

Unfortunately, any data presented at this meeting is not publicly available, though a summary will be provided by IRSF at a later date, so we will have to wait and see what discoveries and improvements/side effects have been seen in the long-term data. I know that families are very curious about what families have seen, especially after being on Trofinetide/Daybue for years now.


On June 7, Dr. Jim Youakim was the keynote speaker. He gave a historical presentation about Trofinetide’s development, with an emphasis on the LAVENDER study and challenges Acadia faced during the Pandemic. This is an area that I know a little bit about and I have to say what an incredible job Acadia did. Against almost insurmountable odds they got creative, bent over backwards, came up with all kinds of creative solutions and got that trial done on time. Even under the best of circumstances not all trials end on time.

A little late, but Rett syndrome waits for no blog!