Top Tweets for #npc1
Exciting news for the NPC community as Apertura Gene Therapy and the NIH advance early-stage gene therapy research for NPC1, bringing new hope to affected families and the future of treatment. Learn more at https://t.co/tLvXywk5VR
#NiemannPick #NPC1 #RareDisease #GeneTherapy
Lawsuit Reveals "Cruel" Experiments on Kittens Continue Under Kennedy and Trump https://t.co/YP6TYnqy63 via @DBrozeLiveFree #RidglanFarms #RFKjr #DonaldTrump #MAHA #NPC1
#DidYouKnow #NiemannPick disease is a #RareDisease, affecting fewer than 9,000 people worldwide. Join us this #RareDiseaseDay on February 28 to support the Niemann-Pick community and help raise awareness.
$RFL #NiemannPick #NPC1
Honored to attend the Firefly Fund Dreams Gala 2026 and support Firefly’s mission in service of the rare disease community.
An inspiring evening alongside others committed to advancing care and improving the lives of patients and families.
$RFL #NPC1 #NiemannPick #RareDisease
Stop by our poster presentation today, from 3:30–5:30 PM PST at #WORLDSymposium 2026:
Intravenous hydroxypropyl-beta-cyclodextrin (HPBCD): Biomarkers, efficacy, and impact of dosing and administration route on therapeutic index
$RFL #NPC1 #NiemannPick #RareDisease
Happening today at #WORLDSymposium 2026 📍
We will be presenting three posters this afternoon from 3:30–5:30 PM PST. Don’t miss the opportunity to stop by, learn more, and connect with our team.
$RFL #WORLDSymposium #RareDisease #NiemannPick #NPC1 #LysosomalStorageDisorders
Very happy with our paper together with the @NoamZelcer lab. Identifying #NPC1 as cargo of the #VPS41 dependent pathway to #lysosomes reveals a tole for LAMP carriers in #cholesterol homeostasis. https://t.co/PNLw2ULKcm
@UMCUtrecht @amsterdamumc
#KCEX Initial Listing! 🎉 Enjoy zero fees on spot trading
#NPC1 is now live on the KCEX Spot Market. @NPCMemeOnSol
🔹 Deposits: Opened
🚀 NPC1/USDT Trading Starts: 2025-12-12 3:40 (UTC)
👉 Trading Now: https://t.co/saCcZsODob
🌐 Details: https://t.co/A21e0KXzAM
#KCEXSpot #KCEXListing
#DidYouKnow: Our TransportNPC™ trial is the most comprehensive pivotal study ever conducted for Niemann-Pick Disease Type C1, spanning global reach, diverse patient participation and meaningful clinical outcomes.
$RFL #NiemannPick #NPC1
We’re united by a shared purpose, to raise awareness and improve the lives of patients, families and caregivers impacted by rare and serious diseases. Our commitment is unwavering, and the progress we’re making is real.
$RFL #NiemannPick #NPC1
At Cyclo Therapeutics, our mission is driven by compassion and purpose. We stand with patients, families and caregivers affected by rare and serious diseases, working every day to bring hope, progress and meaningful change.
$RFL #NiemannPick #NPC1
This #NiemannPickAwarenessMonth, learn how patient registries drive research and improve lives. Visit https://t.co/Mx0TVKLf5s
to get involved.
$RFL #NiemannPick #NPC1 @inpdr_registry
#DidYouKnow: Niemann-Pick Disease Type C is a rare, progressive, and fatal genetic disorder. A defect in the NPC1 protein causes cholesterol and lipids to build up in cells, leading to organ dysfunction and cell death.
$RFL #NiemannPick #NPC1 #RareDiseaseAwareness
Niemann-Pick Disease impacts ~1 in 100,000 people, with about 35 new U.S. cases each year.
Learn more about #NPC1: https://t.co/osRXB2QmH7
$RFL #NiemannPick #NPC1
Let’s keep raising awareness for #NiemannPick
Learn more about #NPC1 and how you can get involved: https://t.co/U5nwWQxW0D
$RFL #NiemannPickAwarenessMonth
October is #NiemannPickAwarenessMonth
Join us in raising awareness for Niemann-Pick Disease and supporting those affected. Learn more at https://t.co/TtMmgfQTwA
.
$RFL #NiemannPick #NPC1 @nnpdf
We are driven by the desire to deliver meaningful therapeutic options for patients in need.
https://t.co/pZ9VJQuQI7
$RFL #NiemannPick #NPC1
Rafael Holdings Announces Presentation of Preliminary Data from Ongoing Phase 3 TransportNPC™ Open-Label, Single-Arm Sub-Study in Patients Less Than 3 Years Old
https://t.co/xaPIVbXCLN
$RFL #NiemannPick #RareDisease #NPC1
Sign up for email alerts and be the first to know about company news, clinical milestones and more!
https://t.co/4ZalBitKu6
$RFL #NiemannPick #NPC1
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