Mila's Miracle @stopbatten
Mila fought hard against Batten disease, a rare fatal condition with no cure. Her story is now giving new hope to millions with genetic disease. stopbatten.org Boulder, CO Joined February 2017-
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Ethan Perlstein @eperlste
17K Followers 2K Following ceo @PerlaraPBC (@ycombinator W16), ceo @epalrestat, solo vc @AngelList, evo pharmacologist, mTOR worshipper, HODLer since '14, cofounder of Kai & Luca, bio/acc
Effie Parks @OnceUponAGene
5K Followers 3K Following Nuclear Advocate | Award Winning Podcaster | Speaker | Captain Connection | #RareDisease #RareMama to my sweet, Ford, who lives with #CTNNB1 🦓
n-Lorem Foundation @n_lorem
1K Followers 572 Following Discovering, developing, and providing personalized experimental ASO medicines to treat nano-rare patients — for free, for life
Luke Rosen @lukebrosen
2K Followers 432 Following Dad of 2 amazing kids. Founder of https://t.co/M8dxuYqmgL. Works with families affected by rare neurological diseases and cancer. Baseball and hockey. Firefighter.
Kelsie Snow @kelsieswrites
61K Followers 1K Following Storyteller, writer, podcaster @sorryimsadpod, ALS advocate, widow, mom, dual citizen (🇨🇦🇺🇸). Instagram & TikTok: kelsieswrites
Dr. Tracy Dixon-Salaz.. @TracyDixonSalaz
3K Followers 974 Following Mom. Rare Disease & LGS Advocate. Neuroscientist. The largest most neglected healthcare resource, worldwide, is the patient. Executive Director @LGS_Foundation
SynGAP Research Fund .. @cureSYNGAP1
10K Followers 6K Following #SYNGAP1 🧬 = 🧠NDD DEE causing #Epilepsy #Autism #ID #Sleep #GI. Incidence = 6️⃣.1️⃣/💯k ICD10 https://t.co/4UJNF5bKTl https://t.co/pjCrzqlbV7 🎙
AnnemiekeAartsma-Rus @oligogirl
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Dr. Heather Mefford @hcmefford
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Fyodor Urnov @UrnovFyodor
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Beacon for Rare Disea.. @RareBeacon
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Adam Johnson - DadVoc.. @RareDiseaseDad
1K Followers 1K Following Dad w/#RareDisease | #Mito Advocate |🎙Host: #ParentsAsRare | Educator | Support | Kindness | Dad Jokes |#MentalHealthMatters | #BoiseState | ⚾️ @Cubs 🏈@49ers
Alok Tayi @aloktayi
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Clement Chow @ClementYChow
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Stephanie Fischer @RarePOV
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Gemma Carvill, PhD @CarvillLab
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Heidi Grabenstatter @PatientIntv
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AstridBunyan @7r6hNJQ8cTb8s8U
0 Followers 37 Following
Jillian Wise,PhD @jillianmcwise
281 Followers 541 Following Cancer Immunologist and Computational Biologist, PhD. Women in Science. Travel Enthusiast. Mother. Wife.
EveryONE Medicines @EveryONEMed
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Erica Cischke 🧡 @e_cisc
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Steph Hughes @NclSteph
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Katie Waller @K_A_Waller
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NCL Database @NclDatabase
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Sinéad McGlacken-Byr.. @SMCGB
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Genomics England @GenomicsEngland
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Stolina Qirjazi APRN @SQirjazi
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ALS Advocacy @alsadvocacy
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Julia Brown, PhD @juliaehbrown
1K Followers 2K Following Appreciates conversations where both things can be true. | Anthropologist & bioethicist @UCSF
Gabriela Arguedas😷.. @gaby_arguedas
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Richard Coca @RichardACoca
217 Followers 255 Following 1st year 🧠 PhD student @NeurosciBU, 👨🎓MS ‘23 @StanfordMed, BS ‘22 @Stanford
Mayo Clinic Center fo.. @MayoClinicCIM
6K Followers 2K Following The Center for Individualized Medicine integrates the latest in genomic science, precision medicine into clinical practice at @MayoClinic. RTs ≠ endorsements.
Radhika Dhamija, MD, .. @NeurogenesDr
817 Followers 432 Following momX2, Geneticist&pedsneuro @mayoclinic,Med school @aiims_nd
Simons Searchlight @s_searchlight
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Pat Sharp @PatSharpChem
542 Followers 1K Following Scientific co-founder Gate Bio | curing disease | prev: @Merck medicinal chemistry
Dutch Antisense Thera.. @Dutch_Antisense
23 Followers 61 Following DATS is designed to connect young investigators in the field of antisense therapeutics to strengthening the field in the Netherlands 🇳🇱 and beyond 🏳️🌈
Carol @carolrivera76
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CUPA Working Group @CUPAethics
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Kathleen Helen Burns @KathleenHBurns
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Frauke Coppieters @CoppietersF
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Mary @maryozaeta45
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Sudeshna Manna @sudimanna
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Love To Emma @Lovetoemma
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Alessandro Ramsey @Alessandro75345
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Reese Caldwell @reesecald
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Letizia Vestito @letizia_ve
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Nelesoson @nelesoson48584
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Simon Quayle @TheSimonQuayle
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Patrice Milos @milospm1206
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Malcolm Vosen @abacus_agent
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Brian Wallach @bsw5020
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n-Lorem Foundation @n_lorem
1K Followers 572 Following Discovering, developing, and providing personalized experimental ASO medicines to treat nano-rare patients — for free, for life
SynGAP Research Fund .. @cureSYNGAP1
10K Followers 6K Following #SYNGAP1 🧬 = 🧠NDD DEE causing #Epilepsy #Autism #ID #Sleep #GI. Incidence = 6️⃣.1️⃣/💯k ICD10 https://t.co/4UJNF5bKTl https://t.co/pjCrzqlbV7 🎙
AnnemiekeAartsma-Rus @oligogirl
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CACNA1A Foundation @cacna1a
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Fyodor Urnov @UrnovFyodor
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Beacon for Rare Disea.. @RareBeacon
12K Followers 11K Following Beacon is a UK-based charity that is building a united rare disease community with patient groups at its heart. Previously known as Findacure.
Matthew Herper @matthewherper
111K Followers 3K Following Writing and events at STAT. This is biology's century; Every data point has a face. ‘21 Polk Award. Mastodon: @[email protected]
Jon Watts @jwattsgroup
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Oligonucleotide Thera.. @OTSociety
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Ana Mingorance @CNSdrughunter
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EveryLife Foundation @EveryLifeOrg
7K Followers 3K Following Nonprofit org. dedicated to advancing the development of treatment & diagnostic opportunities for rare disease patients through science-driven public policy.
BDSRA Foundation @BDSRA
1K Followers 213 Following Batten Disease Support, Research, & Advocacy. Together, we are #BattenAdvocatesForACure. Donate here: https://t.co/soo8KxPl7I
International SCN8A A.. @SCN8AAlliance
763 Followers 467 Following The International SCN8A Alliance is working collaboratively with families, clinicians and researchers to advance the understanding of and treatments for SCN8A.
Perlara @PerlaraPBC
5K Followers 3K Following https://t.co/fyNyv0FS57 The first biotech public benefit corporation est. 2014. Building the YC for Rare. Co-owner @epalrestat
Taylor Kane @rarelikeher
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BBC Sounds @BBCSounds
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University of Oxford @UniofOxford
1.0M Followers 2K Following Welcome to our official account 👋 Follow for the latest news, research and updates about life at Oxford.
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Oxford-Harrington Rar.. @OHRareDisease
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ABPI @ABPI_UK
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MHRAgovuk @MHRAgovuk
32K Followers 593 Following We want our social channels to be safe spaces for healthy, open and insightful discussion. Please read our community guidelines here https://t.co/vloFQ9oujb
The Guardian @guardian
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12K Followers 1K Following Columnist @WashingtonPost @PostOpinions. Author, The Optimist’s Telescope. Former Boston Globe Ed Page Editor, WH advisor, MIT, NYT. First one in the water.
Caroline Seydel @CarolineSeydel
761 Followers 1K Following Science writer covering genetics, cancer, biotech. "Truth is a matter of the imagination. The soundest fact may fail or prevail in the style of its telling."
nature @Nature
2.6M Followers 4K Following Research, News, and Commentary from Nature, the international journal of science. For daily science news, get Nature Briefing: https://t.co/wGmQlQ8a4D
Wheelers Warriors @WheelersWarrior
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Judy Stecker @Judy__Stecker
909 Followers 1K Following Wife of @courtneystecker - Mom of @WheelersWarrior- @HHSGov alum, @SyracuseU and @Georgetown alum -GPhiB- views=own RTs not endorsements
Screen4Care @Screen4Care
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Medscape @Medscape
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RARE-X @RARE_X_
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Chris Wyant @chwyant
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Genomics England @GenomicsEngland
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David Leonhardt @DLeonhardt
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Bill Clifton, MD @docclift
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TBRS Community @TBRSCommunity
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Natasha Loder 🐋 @natashaloder
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Holly Carmichael @CarmichaelHolly
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Kristin Kantautas @wolfeofscience
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Sam Finlayson @IAmSamFin
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OpeTreatments @OpenTreatments_
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Ultragenyx @ultragenyx
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Jocelyn Kaiser @jocelynkaiser
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AllStripes @_allstripes
3K Followers 1K Following Our mission is to unlock new treatments for people affected by rare disease.🚀
GV @GVteam
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Scott Gottlieb, MD @ScottGottliebMD
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New art project: the concept is to carve an image into wood in a semi-abstract form that allows the viewer to both recognize and personalize the image. The image is carved as an array of precisely cut dimples in a piece of pale wood pre-coated with black paint. 1/7
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#RareDiseasesCampaign2023 is out today 🧬 Grab your copy in today's @guardian and online at ow.ly/IrB630sxhCk featuring Julia Vitarello with EveryONE Medicines #RareDiseaseAwareness #ResearchForRare #SupportForRare
And lastly among the Girls and #Women4Screen4Care we want to thank today 🙏 Mila and Julia Vitarello for inspiring us to change how we think about curing rare disease, and do it so passionately. Where would we be now without you? #WomeninScience #February11 #Screen4Care
Catch this great line-up at #DiscoveryForum on 22 Nov: Dr @timyu and Julia Vitarello (@BostonChildrens @stopbatten) Prof @manoliskellis (@MIT) Nathan Lawless (@Boehringer_UKI) James Hadfield @coregenomics (@AstraZeneca) Online places still available: discovery-forum.co.uk
What a powerful story from Julia Vitarello (@stopbatten) about her journey to find answers about her daughter, Mila's, rare condition. A moving reminder of the greater purpose we're working toward. #discoveryforum Read more here: milasmiracle.org
Phenomenal people (Julia Vitarello) pushing through heartbreak and scientific/therapeutic/regulatory barriers to pave the way for platform-based drugs and personalised genomic @GenomicsEngland @stopbatten @timyu #DiscoveryForum #RareDisease #FromMilaToMillions
We’re at the @GenomicsEngland #discoveryforum. The moving and inspiring account of Mila's life from her mother Julia Vitarello @stopbatten and clinician scientist @timyu on how whole genome sequencing brings hope for the millions of children with rare genetic diseases
Very poignant testimony by Julia @stopbatten & how to find personalised treatment for many more #RareDisease #patients Timing and scaling up will be key #DiscoveryForum
What a powerful story from Julia Vitarello (@stopbatten) about her journey to find answers about her daughter, Mila's, rare condition. A moving reminder of the greater purpose we're working toward. #discoveryforum Read more here: milasmiracle.org
Learn more about our efforts to accelerate research by supporting collaborations between patients and researchers through our #RareAsOne Network RareAsOne.org
We’re also supporting collaborative teams examining the pathophysiology and mechanistic underpinnings of neurodegenerative #RareDisease to break down scientific silos + advance our understanding of health chanzuckerberg.com/patient-partne…
When patients are partners in biomedical research, we accelerate science and the path towards treatments + cures for #RareDisease. Our newest grantees are working to address critical gaps in pediatric & neurodegeneration research 🧵
More deserves to be said about that but for now, grateful to @nytimes for sensitive coverage re: new potential new toxicity mode of some high-dose intrathecal ASOs, impacting 2 families in an accelerated n=2 trial. @OTSociety @Erika_Check nyti.ms/3zn7k01
Not all important news to share is good. This week at the American Neurological Association in Chicago, we shared how, beginning in 2018, we began studying ASO therapeutic strategies for one of the most devastating infantile epilepsy syndromes we know. @TheNewANA1 #ANA2022
@timyu @TheNewANA1 Thanks for sharing this difficult news for all involved. Only if we share and are transparent can we move forward. Thanks for setting the example for the N=1 field (again)
Ok, so my amazing group (I presume!) foil-wrapped my entire office, and everything in it, to celebrate my just-announced promotion to professor.
This week on The G Word, hear the powerful story of finding answers for Mila, who was diagnosed with Battens Disease: ow.ly/BYHN50KCa5i Her mother, Julia, and her doctor, @timyu, join our Chief Medical Officer, @Rich_Genomics. @stopbatten
what’s the science behind pizza tasting better when frank sinatra is playing
Excited to share the Multicentric Carpotarsal Osteolysis/MCTO Cure Roadmap commissioned by @sophiesneighbor perlara.substack.com/p/mcto-cure-ro…
Our final Batten fact is a reminder to everyone affected by Batten that you are not alone, and the Batten community’s presence is felt all over the world. Thank you for raising awareness with us! See the link in bio to register for the Virtual 5K! #BattenDay2022 #BattenAwareness
Nothing about us without us!
@cziscience Rare As One is the synergy the rare disease community needs to move treatments forward. What an incredible group of people I got to hang out with and hug all week! More power to everyone searching for treatments! #RareAsOne #PatientLedResearch #RareDiseaseTruth
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