RASopathies Network
@rasnet.bsky.social
Nonprofit research advocacy org for conditions caused by dysfunctional RAS/MAPK signaling. #rasopathies #raredisease by Stronach in Pittsburgh
Dr. Stieglitz will study daraxonrasib in JMML (juvenile myelomonocytic leukemia). JMML is associated with certain PTPN11 variants in Noonan syndrome. #pediatrics #oncosky lnkd.in/p/eDZZ32-x
Daraxonrasib: A Promising Cancer Drug for 2026 | Elliot Stieglitz posted on the topic | LinkedIn
What is the most important cancer drug of 2026? Hands down, that's daraxonrasib. A first-in-class, direct inhibitor of RAS (previously thought to be impossible) that has transformed the field of panc...
lnkd.in
#RASDialogueBlog Advancing RAS/RASopathies Therapies @NCI ‘s Frederick National Lab 🧪 #medsky #pediatrics #oncosky www.linkedin.com/posts/freder...
Rethinking rare disease: How the ART Initiative is connecting genomics, the clinic, and the lab bench for RASopathies | Frederick National Laboratory | Frederick National Laboratory for Cancer Researc...
What happens when you build a collaboration on the foundation of connecting population genomics, longitudinal patient follow-up, and laboratory models into one continuous loop? In this month's #RASDia...
linkedin.com
#RASDialogueBlog Advancing RAS/RASopathies Therapies @NCI ‘s Frederick National Lab 🧪 #medsky #pediatrics #oncosky www.linkedin.com/posts/freder...
Rethinking rare disease: How the ART Initiative is connecting genomics, the clinic, and the lab bench for RASopathies | Frederick National Laboratory | Frederick National Laboratory for Cancer Researc...
What happens when you build a collaboration on the foundation of connecting population genomics, longitudinal patient follow-up, and laboratory models into one continuous loop? In this month's #RASDia...
linkedin.com
Wow, That's stark. I'd like to see some more funding for science and clinical trials for treatments.
BWJones@bwjones.bsky.social · 7d agoYou, personally are spending, on average ~$2,500 per person that the United States is deporting. We could fund Medicare for 12 Million Americans with that national budget. By the way, you spend only ~$139 a year on biomedical research through the NIH. www.epi.org/publication/...
🙌🏻From the Spence lab. We hope these expt models will shed light on GI problems experienced by kids with RASopathies. What is RAS/Mapk doing in all those gut and intestinal cell types? Maybe its the confluence of muscle, neuron, lymphatic, endocrine, and endothelial dysfunction. 🤷🏼♀️💩 🧪
Nature Cell Biology@natcellbio.nature.com · 2w ago☕Johnson et al. combine single-cell and spatial transcriptomics approaches to build a spatially resolved atlas of the developing human intestine and intestinal organoids, uncovering spatial organization and fibroblast heterogeneity. www.nature.com/articles/s41...
Clinical implications of RAS mutations in AML: prognostic significance is based upon the involved gene and mutation complexity ashpublications.org/bloodadvance...
Clinical implications of RAS mutations in AML: prognostic significance is based upon the involved gene and mutation complexity
Key PointsComplex mutations in KRAS predict poor outcomes in AML; complex NRAS and KRAS mutations confer an inferior prognosis in KMT2A-r AML.Noncomplex KR
ashpublications.org
🙌🏻From the Spence lab. We hope these expt models will shed light on GI problems experienced by kids with RASopathies. What is RAS/Mapk doing in all those gut and intestinal cell types? Maybe its the confluence of muscle, neuron, lymphatic, endocrine, and endothelial dysfunction. 🤷🏼♀️💩 🧪
Nature Cell Biology@natcellbio.nature.com · 2w ago☕Johnson et al. combine single-cell and spatial transcriptomics approaches to build a spatially resolved atlas of the developing human intestine and intestinal organoids, uncovering spatial organization and fibroblast heterogeneity. www.nature.com/articles/s41...
HuR degradation reveals dependencies in BRAF-driven cancers: Trends in Cancer www.cell.com/trends/cance...
HuR degradation reveals dependencies in BRAF-driven cancers
Targeting RNA-binding proteins has been shown to be challenging due to the absence of druggable pockets. Recently, Lu et al. identified a molecular glue capable of degrading HuR and suppressing BRAF-m...
cell.com
Cincinnati Children's has received a six-year, $38.4M NIH grant renewal to lead the next phase of the Bench to Bassinet Program. The new initiative, B2B CHANGE, will connect genomic discoveries to long-term health outcomes and future care needs. Congratulations to the team behind this work!
$38.4 Million NIH Grant Renewal to Expand Landmark Congenital Heart Disease Genomics Program - Research Horizons
Cincinnati Children’s receives a $38.4 million NIH grant renewal to expand Bench to Bassinet, a national congenital heart disease genomics program.
scienceblog.cincinnatichildrens.org
🗣️ New @ucsfchildrens.bsky.social study in @ajpmonline.bsky.social! 🧠Adolescents in the low-to-moderate and moderate-to-high problematic social media use trajectories had lower cognitive performance 🔗 Read here: tinyurl.com/socmind @theabcdstudy.bsky.social
A major US trial finds silver diamine fluoride can effectively stop severe tooth decay in young kids - offering a quick, noninvasive alternative to complex dental work! 🦷✨ www.news-medical.net/news/2026080... #Pediatrics #Dentistry @jamapediatrics.com
Large US trial supports silver diamine fluoride for severe early childhood caries
A phase 3 randomized trial of 830 young children with severe early childhood caries found that 38% silver diamine fluoride arrested 54.0% of eligible cavitated dentin lesions at six months, compared w...
news-medical.net
Today in @cp-cell.bsky.social, researchers led by @ucsanfrancisco.bsky.social's Ethan Winkler, MD, PhD, publish the most comprehensive cell atlas of the brain’s blood vessels, revealing how a cell’s location influences vascular disease in the brain. 🧪 🧠📈 www.cell.com/cell/fulltex...
Job Syndrome, or Hyper-IgE Syndrome, is a rare STAT3-linked immunodeficiency causing recurrent skin and lung infections, chronic eczema, and elevated IgE levels from infancy. Learn more: https://dub.sh/HIES You may be rare, but you're not alone! #rareDisease #rareDiseaseAwareness
Kawasaki Disease is an acute illness causing blood vessel inflammation in young children, marked by persistent fever and rash, that can damage the heart if left untreated. Learn more: kdfoundation.org You may be rare, but you're not alone! #rareDisease #rareDiseaseAwareness
An advanced wearable with motor sensors captures growth metrics and motor growth charts in infants playing at home over the course of more than a year—offering an unparalleled ability to track early patterns in motor and neurodevelopment. #ScienceTranslationalMedicine https://scim.ag/4x31bBS
At-home wearable measurements provide detailed growth charts of infant early gross motor development
Evaluation of infants’ spontaneous motor activity with at-home wearables helps construct detailed growth charts of early gross motor development.
scim.ag
Online Now: Mechanism of MEK1 phosphorylation by the N-terminal acidic motif-mediated asymmetric BRAF dimer Online now:
Mechanism of MEK1 phosphorylation by the N-terminal acidic motif-mediated asymmetric BRAF dimer
Kondo, Notbohm, et al. show that the BRAF N-terminal acidic motif engages the dimeric interface to drive an asymmetric arrangement in which only one protomer adopts a canonical active kinase conformation. Captured MEK1 Ser222 phosphorylation and mutational analysis corroborate that this motif is required for full BRAF kinase activity.
dlvr.it
What a surprise - a tiny, 1 mm long planktonic bunny 🐰. This a chaetopterus worm larva. Kattegat, The North Sea 🌊 🔬 Chaetopterus, known as the parchment worm, is a highly specialized marine polychaete living the adult life inside self-constructed, papery tubes that resemble parchment paper.
Build platforms that can be applied to n of 1
Kathleen Bachynski@bachynski.bsky.social · 4w ago“The center is not going to solve the problem of getting genetic therapies to patients overnight, its founders emphasized. But the researchers hope the work will lead to methodologies that other doctors can pick up and apply… To achieve that, the center will make its work public whenever possible.”
Delighted to see the latest preprint progress to publication with Advanced Science. We built the protein FRET ruler as a bridge between biophysicists and cell biologists - realising the potential of FRET link measurements from molecules to cells. advanced.onlinelibrary.wiley.com/doi/10.1002/...
We're thrilled to announce the Center for Therapeutic Genetics (CTG) — a new collaboration between JAX, @broadinstitute.org, and @bostonchildrens.bsky.social to develop genetic medicines for rare and ultra-rare diseases. 🔬🧪🧬 go.jax.org/Center-Therapeutic-Genetics
Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory launch the Center for Therapeutic Genetics
A new collaboration to develop precision medicines for those with rare diseases, using infrastructure and repeatable practices that will be shared with others.
go.jax.org
How do sarcomeric mutations disrupt the transition to adult cardiac metabolism? Our study in TNNT2-linked HCM hiPSC-CMs reveals an early energetic deficit, paradoxical mTORC1 suppression, and a pathway to therapeutic rescue. Congrats to Homa Hamledari and the team! www.biorxiv.org/content/10.1...
Molecular Rewiring in TNNT2-Linked Hypertrophic Cardiomyopathy hiPSC-Derived Cardiomyocytes upon Metabolic Maturation
Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) are widely used to model cardiac development and inherited cardiomyopathies, yet their immature metabolic state limits interpreta...
biorxiv.org
Build platforms that can be applied to n of 1
Kathleen Bachynski@bachynski.bsky.social · 4w ago“The center is not going to solve the problem of getting genetic therapies to patients overnight, its founders emphasized. But the researchers hope the work will lead to methodologies that other doctors can pick up and apply… To achieve that, the center will make its work public whenever possible.”
In case you missed this Cancer Discovery paper in the news last week: First-in-Human Testing of a Mutant KRAS Vaccine for Pancreatic Cancer Interception in High-Risk Cohorts - by Daniel Haldar, Amanda Huff, Michael Goggins, Elizabeth Jaffee, Neeha Zaidi, and colleagues @aacrjournals.bsky.social
American Association for Cancer Research (AACR)@theaacr.bsky.social · last mo.A vaccine to intercept pancreatic cancer in high-risk individuals was safe and elicited specific and durable immune responses, according to research published in the AACR journal Cancer Discovery. Learn more: www.aacr.org/about-the-aa...
🚨 Now available on Nature Careers ! #Recruitment #Ottawa
Tier 2 Canada Research Chair in Vascular Links to Neurodevelopment - Ottawa, Ontario (CA) job with University of Ottawa, Ontario CANADA | 12861923
uOttawa, in partnership with the OHRI, invites applications for a Tier 2 CRC in Vascular Links to Neurodevelopment and their impact on brain health.
nature.com
What if we could design proteins to precisely control cell behavior? I’m thrilled to share our latest work, led by @kinasekid.bsky.social, @sauravk05.bsky.social, Mia Donald-Paladino, and Dongyang Li, developing de novo miniproteins that activate or inhibit kinases. www.biorxiv.org/content/10.6...
De novo design of selective kinase modulators
Protein kinases are critical regulators of cellular signaling, but precise modulation of their activity remains challenging due to their high structural conservation. Here, we present de novo designed...
biorxiv.org
Frank McCormick is a force of nature. Love that photo!! He spoke at our 2025 symposium and has a longstanding interest in RAS signaling AND RASopathies!!
American Association for Cancer Research (AACR)@theaacr.bsky.social · last mo.Dr. Frank McCormick's research clarifying how KRAS mutations alter RAS signaling helped lay the foundation for the first targeted therapies against this common cancer driver. Read his story in Leading Discoveries: buff.ly/xeuUL3W
