Congratulations to Encoded, Jaguar, Aparito and what will we count? How will we know these therapies are working? #S10e132

SYNGAP10

Congratulations to Encoded, Jaguar, Aparito and what will we count? How will we know these therapies are working? #S10e132

TWO Gene therapy INDs approved in the past week! JAG201 for SHANK3 https://pmsf.org/breaking-news-from-jaguar-gene-therapy/ ETX101 for SCN1A https://encoded.com/press-releases/encoded-therapeutics-announces-us-ind-clearance-and-australian-cta-approva...

February 7, 2024

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What is this SYNGAP1 illness / disease / syndrome / NDD / DEE / MRD5 / NSID actually called? #S10e133

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SYNGAP1 is complex, we need to partner with our clinicians to improve care & get ready for repurposing. #S10e131

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Congratulations to Encoded, Jaguar, Aparito and what will we count? How will we know these therapies are working? #S10e132

TWO Gene therapy INDs approved in the past week!

JAG201 for SHANK3 https://pmsf.org/breaking-news-from-jaguar-gene-therapy/

ETX101 for SCN1A https://encoded.com/press-releases/encoded-therapeutics-announces-us-ind-clearance-and-australian-cta-approval-for-dravet-syndrome-gene-therapy-candidate-etx101/

Mike’s Gene Therapy Cheat Sheet

https://docs.google.com/spreadsheets/d/1jwH5piRH9gOmylz-pCNd_DFnpEHJ6EkFyoaMjidBk6c/edit?usp=sharing

TAKE THE ROCHESTER SURVEY

https://redcap.link/NDDCaregiverSurvey

SPRINT FOR SYNGAP24 is on!

https://Syngap.Fund/Sprint24

APARITO ANNOUNCEMENT

LI – https://www.linkedin.com/feed/update/urn:li:share:7157828674245783552/

X – https://x.com/cureSYNGAP1/status/1751969751621046667

FB – https://www.facebook.com/cureSYNGAP1/posts/pfbid07mabzGJhcZZZkm3vUy9EkZutAtTL16y4gytED52Xyzjayp3ew62zEXiDA8aEsuYWl

This is the program: https://www.aparito.com/patient-group-programme/

RAVICTI GROUP

https://www.facebook.com/groups/butyrate/

Give all three of our podcasts 5 stars everywhere.

https://podcasts.apple.com/us/channel/syngap1-podcasts-by-srf/id6464522917

This is a podcast subscribe!

https://podcasts.apple.com/us/podcast/syngap10-weekly-10-minute-updates-on-syngap1/id1560389818

Episode 132 of #Syngap10 – February 6, 2024

#epilepsy #autism #intellectualdisability #id #anxiety #raredisease #epilepsyawareness #autismawareness #rarediseaseresearch #SynGAPResearchFund #CareAboutRare #PatientAdvocacy #GCchat #Neurology #GeneChat

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