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Give me six hours to chop down a tree and I will spend the first four sharpening the axe.

- Abraham Lincoln

Dear Friend of SRF,

Praxis, a rare disease biotech with a $2B market cap, announced they are launching a natural history study for SCN2A with Ciitizen this morning.

Aside from being big news for our friends in the SCN2A community, it is a strong endorsement of the Ciitizen platform.  SRF was the first rare disease group to partner with Ciitizen ensuring this best in class platform is available to our community.  JR  & Virginie both strongly encouraged every family to join.  Only last month, Mike made the point that when a company has a potential drug they need a natural history study to inform trial design--and if good data isn’t in place then they will have to start one.  And a $2B biotech just did exactly that, with Ciitizen.  

When they or another biotech has a treatment for SYNGAP, they won’t have to start a study.  It is happening right now.  If you are a US family who has not yet signed up, you are welcome Ciitizen.com/SYNGAP1.  International families can join in April.

In other news, the #TeamSRF has events planned for this Friday and next week, we also have two exceptional webinars coming up and some helpful reading on ABA.  Read up and join us--there's so much work to do.  Volunteer &/or donate now.

Time is brain,

Mike

Tony's Dad & Managing Director of SRF 

 

Únase a nosotros a el Primer Congreso Científico Virtual SYNGAP1 en Español. #SRFCC1

Friday is #SRFCC1.  This Friday our leaders of Latin America are hosting the first annual Scientific Congress of SYNGAP1 entirely in Spanish.  There is an incredible lineup of twelve speakers who will share their knowledge and take questions.  We are grateful to Vicky & Marta for their leadership-our community is lucky to have both of them.

 

SYNGAP1 ABA: Elemy Yebe, BCBA

Many in our community know Aaron and Monica Harding - Jaxon’s parents & anchors of our community in California.  If so, you’ve certainly heard about their incredible ABA team under the leadership of Elemy Yeme, MEd, BCBA.  We are fortunate that Elemy is sharing her deep knowledge of ABA for Syngap.  Please ready her article on ABA & face masks and sign up for her #SRFwebinar in March.

 

SRF Webinars keep getting better

Speaking of Webinars, the recent session with Prof. Kind of the PWC at University of Edinburgh was excellent.  If you missed it, make sure to watch it on the Webinars page. You will have a better understanding of how research is done with animal models.

Looking forward, we have Prof. Ahituv speaking in April.  It promises to be exceptional as well.

 

Next Weekend: US & UK Caren Premiere

We can’t wait to share this movie with you.  Sign up here if you want to join us on

  • Friday Fed 26th at 4:30 PST,  or
  • Sunday Feb 28th #RareDiseaseday for a UK/Euro session at 7:30 PM GMT. 

After the movie, you will have questions, and we will have Caren’s sister, the Director, SYNGAP parent Jess Duggan & other SRF leaders there to discuss. 

Today we are releasing this interview with Jess about her experience making the movie.  It’s good.  Also, don't miss Caren’s #Warrior Wednesday profile by her sister Nancy.

Have you seen the trailer?

 

Remembering Mila

The Rare Disease community has many heroes & heroins.  Julia Vitarello is a giant among them.  When her daughter was diagnosed with Batten’s disease she changed our world by developing a bespoke therapy for Mila in record time, a drug called milasen. The drug gave Mila more time, but last week we learned that her spirit left the body--breaking the hearts of all of us who knew her story or had met her mother. 

As a community, we send Julia and her family our love and support.

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SynGAP Research Fund
1270 Lincoln Avenue
Palo Alto, California 94301
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Mike@syngapresearchfund.org

 
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