Neuren Pharma announces agreement with FDA on #PhelanMcdermidSyndrome co-primary endpoints for Phase3:
➡️ Receptive communication (measured with Vineland)
➡️ Global improvement (measured with PMS-anchored CGI)

Phase 3 to start mid-2025 ⭐

www.neurenpharma.com/showdownload... #PMS #SHANK3

Check out our "Gene therapies for neurogenetic disorders" review at @cp-trendsmolecmed.bsky.social

So fun to write!

We talk a lot about #RareDisease patient foundations and clinical trial readiness because it matters a lot to us 💜🦓

www.sciencedirect.com/science/arti...

FDA employees had to withdraw last week from external meetings, including the joint FDA-ASGCT workshop on AAV gene therapies. This has also impacted the PFDD meeting on Angelman syndrome.

I'm hoping this "temporary pause" is short-lived. It is already hurting #RareDisease medicine development 💔

Looking forward to getting into LAX early to catch up with the traditional #SYNGAP1 Conference by @curesyngap1.bsky.social and the #Dravet Roundtable by @curedravet.bsky.social both on December 5th just before #AES2024

Who else is coming? 💚

🧬 If you work on a neurological #RareDisease you should be following the news around Neurogene's gene therapy for #Rett syndrome.

They have very positive results in the clinic and also a really bad safety problem at a higher dose. Good chat with CEO in link: ir.neurogene.com/events/event...

Moving over to Bluesky to keep talking about treatment development for #RareDiseases, #epilepsy, and neurodevelopmental diseases in a more friendly environment 🦋

Join me if that’s also your community 🧬🦓