Transcripta Bio retweeted
Honored to be working with @cureSYNGAP1 on drug discovery for SYNGAP1 related disorders with @rarebasepbc. Thanks @JMGraglia and @SYNGAP1mom for all you do for the community!
Read & share this @Newsweek story profiling SRF, our founders (@SYNGAP1mom & @JMGraglia) and Tony's diagnostic journey.
newsweek.com/my-son-syngap1-…
FYI @StanfordChild @IDreamofGenes @Invitae @Labcorp (MNG)
Transcripta Bio retweeted
“These things don’t have to be lonely crusades, but they are. Everyone is on their own right now.”
@onnofaber and @rarebasepbc are a mission to remake the dysfunctional rare disease research space.
buff.ly/3CoXeuP
Transcripta Bio retweeted
We are honored at @rarebasepbc to be working with so many patient communities to create a path forward to find therapies for rare genetic diseases.
Thanks to HOPE4HARPER (hope4harper.com) for enabling our work on #CDKL5 related disorders.
A new article by @illumina shares Jainu and Shruti Jogani’s quest to find therapeutic opportunities for their daughter, Reyna, who is affected by CDKL5 deficiency disorder. The Rarebase Function™ platform is helping to advance their search.
illumina.com/company/news-ce…
Transcripta Bio retweeted
While working to understand not only his own rare disease but also how the broader healthcare industry develops treatments for rare diseases as a whole, Onno Faber uncovered the building blocks of @rarebasepbc. hubs.la/Q01nmD8L0
Transcripta Bio retweeted
👏 And @GraceRubie's story about @onnofaber's efforts to champion solutions for sufferers of #rarediseases won 3rd place in the Consumer category.
neo.life/2021/09/the-champio…
Transcripta Bio retweeted
I'm discussing “🧬 Gene Fixers 🛠: Rare Entrepreneurs” with @eperlste, Julia Vitarello, @vancanwin, and The Gene Fixers. Today, Mar 17 at 3:00 PM EDT in @clubhouse. Join us! clubhouse.com/event/m26pk0W4…
Transcripta Bio retweeted
A #raredisease family (not #SYNGAP1) working with @rarebasepbc just like @cureSYNGAP1 to help their son Landon. I know this incredible mom. Watch this space.
Thanks to @Micah_walker701 of @DispatchAlerts for such a beautiful article. More journalism like this please!
5-year-old Landon has a TBCD mutation.
Allison Bradbury, a principal investigator in the Center for Gene Therapy @nationwidekids said TBCD plays a crucial role in neural development. Because of this, Landon is unable to walk and has difficulty talking.
dispatch.com/story/lifestyle…
Transcripta Bio retweeted
Replying to @transcriptabio
@rarebasepbc is a public benefit corporation that uses genomics to identify treatments for rare diseases. The firm’s unique model relies on small foundations and patient advocacy groups to acquire both funds and patient data. Simone Ellin reports: bit.ly/3bIcw2q
Transcripta Bio retweeted
#HNF collaborates with Rarebase on a Drug Discovery Platform to develop treatments for Charcot-Marie-Tooth (CMT) hnf-cure.org/research/hnf-co… @rarebasepbc #cmtwegotthis #charcotmarietoothdisease #cmtneuropathy #cmt #neurotwitter #rarebase #charcotmarietooth
Transcripta Bio retweeted
ANNOUNCEMENT: More information about our new drug screening partnership with @rarebasepbc to find treatments for Cockayne Syndrome in addition to gene therapy. #CureCockayne
Press Release: riaanresearch.org/downloads/…
Transcripta Bio retweeted
We are so proud to be working with @rarebasepbc and @onnofaber - their incredible co-founder and CEO - to try and find potential drugs that may help treat Cockayne Syndrome.
“We’re turning their efforts into tiny, virtual biotechs.”
@onnofaber—who has a rare disease himself—is uniting people with rare diseases to pool their data & investments to participate in pharmaceutical research.
The goal? Effective treatments & cures.
neo.life/2021/09/the-champio…
Transcripta Bio retweeted
HNF collaborates with Rarebase on a Drug Discovery Platform to develop treatments for #CharcotMarieTooth (CMT)
“We’re thrilled to be collaborating with the HNF to help find treatments for patients with CMT. #cmtwegotthis
Read more: hnf-cure.org/research/hnf-co…
Transcripta Bio retweeted
Thank you @rarebasepbc! Currently driving through CO, 63% through our drive, with ~$61k raised to our $100k goal! Sponsor $1:$1 matches have started! Tune into our stream, donate & share! Live-streaming straight until we reach Redondo Beach tomorrow night! justgiving.com/fundraising/u… – at Colorado, USA
UFD's Cannonball for the Cure
Help UFD Tech raise money to support Syngap Research Fund
justgiving.comSuper proud to collaborate with @cureSYNGAP1 on treatments and cures for #syngap1!
The @curesyngap1 team is currently driving across the country to raise awareness and funds for their kids. Learn more about Cannonball for a Cure #ufdcure
Transcripta Bio retweeted
Replying to @UFDTech
@UFDisciple + @phalliburton + Kevin + @Tesla
🚗Driving 🗽➡️🌇
⏱𝟔𝟎 hours
🤳🏼Livestreamed
=
💰 via @JustGiving
⬇️
@cureSYNGAP1 🧬 501(c)(3)
⬇️
@rarebasepbc
⬇️
#DrugDiscovery 💊
⬇️
Kids with #SYNGAP1 doing better.
Check it out at Syngap.Fund/CBall
@elonmusk #UFDcure
This kid right here.
I love him so much & I'll fight as hard as I can to get him cured.
I'm so thankful we live in a time where that's even a possibility.
Next week, we drive from NYC to LA to raise funds & awareness.
Cannonball for the Cure is gonna be epic.
#UFDCure
Transcripta Bio retweeted
🎙Ep. 27 of #SYNGAP10 w/ @JMGraglia is up!
💊Brief explainer on @rarebasepbc
🏎 Cannonball #UFDcure in 1️⃣8️⃣ days
👟 @BillyFootwear & 🔬 @QStateBio Convos
🏃🏽 Someone is going to win
🎉🗽 GALA in 3️⃣5️⃣ days syngap.fund/gala
Listen and subscribe at syngap.fund/10
Transcripta Bio retweeted
I’m excited and humbled to share our next step in helping to find treatments for patients with rare genetic disorders. Thanks to all our collaborating partners and families, and the amazing team at @rarebasepbc for being part of this to make this happen.
prnewswire.com/news-releases…
Transcripta Bio retweeted
@rarebasepbc launches FUNCTION 🧠 💊🔬 #drugdiscovery w/15 #raredisease orgs.
@ADNPkids is proud to be an anchor ⚓️ partner alongside outstanding groups like @Foxg1Research @curestxbp1 @cureSYNGAP1 @CMTNeuropathy & #Hope4Harper prnewswire.com/news-releases…
Transcripta Bio retweeted
We are thrilled to be an anchor partner of @rarebasepbc new #drugdiscovery platform for #rarediseases called FUNCTION. This groundbreaking platform launches with these incredible rare warriors @cureSYNGAP1 @curestxbp1 @ADNPkids @CMTNeuropathy #HopeforHaper & more
Today Rarebase is excited to launch a neuroscience drug discovery platform in collaboration with 15 rare disease patient organizations! #RareDisease #drugdiscovery #neuroscience
prnewswire.com/news-releases…
Transcripta Bio retweeted
.@Rarebasepbc launches FUNCTION 🧠 💊🔬 #neuroscience #drugdiscovery w/ 1️⃣5️⃣ #raredisease orgs
@cureSYNGAP1 is proud to be an anchor ⚓️ partner alongside outstanding groups like @Foxg1Research @curestxbp1 @ADNPkids @CMTNeuropathy & #Hope4Harper!
prn.to/3EfZFBI #SYNGAP1
Transcripta Bio retweeted
"The meaning and concept of a cure is deeply personal for each patient and their family."
Excellent article by Co-Founder of @rarebasepbc , @onnofaber
rarebase.org/post/what-does-…

























