Personalized Selumetinib Dosing in Pediatric Neurofibromatosis Type 1: Insights From a Pilot Therapeutic Drug Monitoring Study

Advanced Ovarian Cancer Responds to Claudin-Targeted Drug in Preliminary Trial Partial responses in half of patients with platinum-resistant disease, as crowded ADC list grows

Half of heavily pretreated patients with platinum-refractory ovarian cancer responded to an investigational antibody-drug conjugate targeting Claudin 6, a small study from China showed. #AACR26
http://medpagetoday.com/meetingcoverage/aacr/120868

🧪 💊 Pasithea has a cyclized MEK inhibitor , curious to learn more.
How does it compare w first gen MEKi ?
#medsky
#nf1
#rasopathies

Pasithea Therapeutics Announces Grant of Rare Pediatric Disease Designation by FDA to PAS-004 for Treatment of Neurofibromatosis Type 1 - Investing News Network

Our Advocacy Town Hall is tomorrow! Carrie Wolinetz, from Lewis-Burke Associates and former NIH Chief of Staff, will discuss NIH grant distribution, recruiting institute/center directors, and policy issues. Register for FREE: buff.ly/zuo2aHB

#KTTA Pasithea Therapeutics Announces Grant of Rare Pediatric Disease Designation (RPDD) by FDA to PAS-004 for Treatment of Neurofibromatosis Type 1 (NF1)

www.stocktitan.net/news/KTTA/pasithea-thera...

Totally with you! walking around going ah-uh 😄

we have fish crows in pittsburgh.
so are they moving west?

Webinar Reminder: Understanding the FDA's Plausible Mechanism Framework Tuesday, April 21, 1-2 p.m. ET

🧪🧬🩺💊 Unpack the FDA’s new Plausible Mechanism Framework for individualized genetic therapies in NORD’s Rare Action Network virtual webinar. @nordrare.bsky.social #medsky #pedsky
rarediseases.zoom.us/webinar/regi...

As a recipient of federal grants from #NIH (for now! 😭) that funds research in my lab, I'd like to sincerely thank American taxpayers on #TaxDay for investing in scientific research that lays the foundation for medical and technological innovation in this country and keeps us ALL safe and healthy

Rapid melatonin test can help astronauts and others easily monitor their biological rhythm A simple test developed at Washington State University could eventually allow astronauts and others in round-the-clock occupations to monitor their biological rhythms in just minutes using a drop of blood, a paper test strip, and a smartphone-based reader.

A rapid, smartphone-based melatonin test enables precise monitoring of circadian rhythms, offering potential benefits for astronauts and shift workers in environments with disrupted light cycles. doi.org/hbxbjp

Remember to take care of yourselves, parents.
#raredisease #caregiver #medsky

Article titled "Risk of Cardiovascular Disease in Parents of Children Diagnosed With Neurodevelopmental Disorders" by Hui Wang, et al. Study shows increased CVD risk in parents with children having NDDs. Published in JAMA Pediatrics, April 13, 2026.

Parents of children with #NeurodevelopmentalDisorders—including autism, ADHD, or intellectual disability—had a higher risk of developing #CardiovascularDisease, especially with multiple affected children or comorbidities. ja.ma/4sxFnvp

Everylife Foundation for Rare Diseases and Amgen present the #RAREis scholarship fund. Fifty-eight $5000 scholarships are available for individuals with rare diseases to pursue education in Fall 2026. Apply by April 28, 2026 at RareScholarship.org

Amazing opportunity from everylifeorg.bsky.social for the #raredisease community 💙
The #RAREis Scholarship Fund is offering 58 scholarships of $5000 for ppl with RD to help pursue higher education, + access to mentorship & advocacy opportunities
🗓 Apply by April 28
📍 Learn more: RareScholarship.org

Right on!

💊CURE-ID is an FDA/ NIH developed treatment registry to capture rare disease drug repurposing.
#RASopathies patients, caregivers, and clinicians can report experiences using existing drugs (LIKE MEK INHIBITORS) to see what’s working or not.
cure.ncats.io/explore/rare...

#medsky #medical #pediatrics #pedsky #anxiety #mentalhealth

The 9th International RASopathies Symposium The RASopathies are a group of congenital disorders with overlapping clinical manifestations that are caused by pathogenic germline or early somatic variants that result in the hyperactivation of the...

🧪🧬🩺🫀💊 Our conference proceedings are out now! Catch up on the latest work on how aberrant #RAS signaling affects human development.
📌The 9th International RASopathies Symposium - Castel - American Journal of Medical Genetics Part A - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/...

Scholarship opportunity #RAREis
#raredisease
👇🏻

Interesting how the leading edge of the cell front is lifeact-

Elegant work. Do they comment on upper limit to gene size for feasibilty? or maybe just reagents and technicians.

Scholarship opportunity #RAREis
#raredisease
👇🏻

good science 🧬
good neighbors 🤝
good hockey 🐧

Congrats @juliaplank.bsky.social on this fascinating research.
A presentation from Dr. Plank at the RASopathies Symposium last summer describes this work. #neurosky #medsky #neuroscience #myelin #mri
youtu.be/9MQ7FN1e-b0?...

RAS-dependent cancers should be seeing huge improvements with all the therapeutics in the pipeline and combo therapies. What is the data on pancreatic cancer treatments and pediatric brain cancers like HGG and DIPG?

It's not just about "brain power." 🏃‍♂️ Reduced cortical myelin in youth with RASopathies (like #Noonansyndrome) is significantly linked to lower muscle strength and mobility. This is the first study to connect these biological markers to daily physical functioning. 🧬 #RareDisease #HealthResearch

Illustration of cortical sampling across three depths using T1w/T2w ratio images. Figure 1 from linked article:T1w/T2w ratio as an in vivo proxy for reduced intracortical myelin content in youth with RASopathies: a cross-sectional study

New research explores how myelin—the brain's insulation—is affected in kids w/ RASopathies. Using a "genetics-first" approach, Plank et al found widespread ⏬ in cortical myelin, mapping a new path for treatment. 🧠📉🧪
#Neuroscience #RareDisease #MedSky #NeuroSky
OA: pubmed.ncbi.nlm.nih.gov/41905474/

This planet — with all its troubles — is worth caring for.

That should be our single purpose, as a species: To be stewards of this magnificent world, ensuring it thrives long into the future.

85 year old Joan Baez and Jesse Welles at the UC Theatre in Berkeley, CA, March 21, 2026

TT: jessewelles