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

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

FromSynGAP10 weekly 10 minute updates on SYNGAP1


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

FromSynGAP10 weekly 10 minute updates on SYNGAP1

ratings:
Length:
15 minutes
Released:
Feb 7, 2024
Format:
Podcast episode

Description

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
Released:
Feb 7, 2024
Format:
Podcast episode

Titles in the series (100)

Over 900 families are caring for a loved one with the rare disease ”SynGAP” resulting from a variant of the SYNGAP1 gene. This 10 minute weekly podcast is for them. A quick summary of the latest news in the space. The host is Mike Graglia, co-founder & managing director of the SynGAP Research Fund. SRF is a parent-led, all volunteer public charity in the US that strives to accelerate research into treatments for SYNGAP1 so that we can help our loved ones in a timeframe that matters. Learn more at https://www.syngapresearchfund.org/