Category Archives: IRSF

IRSF Scientific Meeting, Daybue and More

Posted on June 19, 2023 by melelllan

The International Rett Syndrome Foundation (IRSF) held its Scientific Meeting on June 5-7, featuring presentations from Acadia, Taysha, Neurogene, and NeuroTec. Anavex did not present as they had no new information to share. Acadia presented LILAC data and introduced a new Real World Efficacy study called LOTUS. Unfortunately, any data presented at the meeting is not publicly available, but a summary will be provided by IRSF at a later date. Dr. Jim Youakim gave a historical presentation about Trofinetide’s development, with an emphasis on the LAVENDER study and challenges Acadia faced during the pandemic. Continue reading →

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IRSF Presents: Parents’ Daybue Trial Experiences

Posted on May 7, 2023 by melelllan

This is a webinar you do not want to miss! Live Parent Panel: My Trofinetide Clinical Trial Experience May 11, 2023 7pm-8pm Eastern time “Join IRSF for a candid Q&A with parents whose daughters participated in different stages of the … Continue reading →

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Acadia Connect is your Connection!

Posted on March 18, 2023 by melelllan

Now that we have accepted that, yes Daybue has been approved, many have already contacted your providers to make sure they will order it. What is next after that??? This is where AcadiaConnect comes in! Once you have been given … Continue reading →

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T-20 Musing: Parents’ Helping Hands

Posted on February 22, 2023 by melelllan

I often see parents who comment on social media that they feel disappointed, or defeated, etc. because they were unable to participate in a clinical trial. But, it takes all types of participation for a trial and ultimate approval (hopefully!!) … Continue reading →

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17 Hopes of September

Posted on September 4, 2022 by melelllan

Our children suffer, greatly, and parents/caregivers are right beside them on that journey. When your child is born you have so many great hopes for them, dreams beyond compare. Then, Rett syndrome, the thief in the night, steals that all … Continue reading →

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