#RAREdisease
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franmartinezgr.bsky.social
Newly identified ARF3 variants strengthen the causal link between Golgi fragmentation and brain malformations #RareDisease #Genetics #morbidgene www.nature.com/articles/s41...
January 9, 2026 at 8:37 AM
rasnet.bsky.social
Right on ! CNBC Cures.
We advocate for a group of #raredisease conditions related to #syngap. the #RASopathies. syngap is a #RAS gap linking it to several other neurodevelopmental syndromes 🧠🧪🧬
🙏 TY for highlighting rare disease stats and stories on your platform
cnbc.com CNBC @cnbc.com · 19h
30 million people are living with a rare disease in the United States, according to the National Organization of Rare Disorders.

That’s why we’re launching CNBC Cures, a new initiative to help raise awareness of rare diseases and improve patient outcomes.

Read more: cnb.cx/4pqqmK7
January 9, 2026 at 1:18 AM
aj-mcknight.bsky.social
Delighted to have contributed to developing UK #RareDisease quality standards within our 4-nation project led by Sue Farrington rarediseaseqs.org

-> draft quality standard for consultation 6-20 Jan 2026 Have your voice heard! #RareDiseaseNI - please add your perspectives on what matters most!
Developing QS for rare disease across the rare disease community
Developing quality statements for rare disease via consensus across the rare disease community.
rarediseaseqs.org
January 8, 2026 at 11:46 PM
aj-mcknight.bsky.social
Thanks to @claire-hill.bsky.social & Dr Grace McMacken showcasing their research in our @qubelfastofficial.bsky.social Molecular Epidemiology & Public Health team-packed RVH room today. #Ageing #RareDiseaseNI #RareDisease #ClinicalTrials #RenalResearchNI #RARDTAC #Neuromuscular #kidney.
Claire presenting ageing biomarkers Grace presenting building diagnostic equity and trial readiness for clinical neuromuscular trials.
January 8, 2026 at 10:42 PM
mgnews.bsky.social
Join the critical discussion: https://bit.ly/4p2z01W

#MyastheniaGravis #MyastheniaGravisNews #Bionews #MGWarrior #ChronicIllness #RareDisease #Neurology #Neurologist
January 8, 2026 at 9:02 PM
mgnews.bsky.social
Humor can lighten heavy days. What made you laugh recently? #RareDisease
January 8, 2026 at 8:38 PM
rarediseaseadvisor.bsky.social
Providing care for patients with Duchenne muscular dystrophy (#DMD) may significantly disrupt caregivers’ employment and productivity, according to a report published in Advances in Therapy

Read more: https://bit.ly/4qIKDM4

#RareDisease #DuchenneMuscularDystrophy
DMD Caregiving Negatively Affects Productivity, Pointing to Need for Better Support
A survey of US-based DMD caregivers revealed the notable effects of caregiving demands on work and productivity.
bit.ly
January 8, 2026 at 7:53 PM
angioedemanews.bsky.social
Taking control of HAE starts at home: https://bit.ly/4ns2bLx

With the right training, self-injecting your medication means faster relief, fewer ER trips, and more confidence in managing attacks.

#Angioedema #HAE #HAETreatment #SelfInjection #ChronicIllness #RareDisease #AngioedemaNews #Bionews
January 8, 2026 at 7:47 PM
angioedemanews.bsky.social
Leave a ❤️‍🔥 in the comments if you have a love-hate relationship with doctors. #RareDisease
January 8, 2026 at 7:46 PM
nmraredisease.bsky.social
🦓 This year, let's make a resolution to raise awareness for #rarediseases not only in #NewMexico but globally for all.

🌶 NM needs a #RDAC!

#nmleg #nmpol #nmrare #nmraredisease #raredisease #showyourstripes #ziazebras
January 8, 2026 at 5:26 PM
rarelife-solutions.bsky.social
At rareLife, we build symposia in partnership with our clients—combining rare compassion, rigorous science, & deep community insight.
From benchmark slides to engaging digital content & discussion,
we help rare science connect & inspire.
www.rarelifesolutions.com
#Symposia #SciComms #RareDisease
January 8, 2026 at 3:05 PM
angioedemanews.bsky.social
#HAE #HereditaryAngioedema #Angioedema #RareDisease #RareDiseaseCommunity #ChronicIllness #ChronicIllnessCommunity #HAEAwareness #HAEWarrior #PatientVoice #PreventiveTreatment #HAEAttack #SwellPride #DisabilityAwareness #InvisibleIllness #RareButNotAlone #BioNews #AngioedemaNews
January 8, 2026 at 3:03 PM
rarectadvocate.com
Melioidosis (Whitmore’s disease) is a rare infectious disease caused by the environmental bacterium Burkholderia pseudomallei, which lives in soil and water in tropical regions.

Learn more: www.melioidosis.info

You may be rare, but you're not alone!
#rareDisease #rareDiseaseAwareness
January 8, 2026 at 3:02 PM
cfnewstoday.bsky.social
Plan smarter travel ✈️ https://bit.ly/3Z2mc0h

Different climates can affect breathing, hydration, and infection risk for people with cystic fibrosis.

#CysticFibrosis #CFNews #CysticFibrosisNewsToday #CFCommunity #CFTravel #RareDisease #LivingWithCF #Bionews
January 8, 2026 at 2:48 PM
gamuts.net
Parathyroid carcinoma https://gamuts.net/x/22266 #RareDisease #MedEd #FOAMrad
January 8, 2026 at 2:01 PM
tisentotx.bsky.social
We’ve completed enrollment in PRIZM, our global Phase 2b study evaluating our investigational medicine’s impact on fatigue and cognitive function in MELAS – a #RareDisease that currently has no approved treatments. #MitochondrialDisease #Biosky www.tisentotx.com/news/tisento...
January 8, 2026 at 12:11 PM
aspenbrave.bsky.social
expectations loom
all day long in human minds
while some roses bloom
with pain from many a thorn
others wither crowns they adorn

©️Aspen
#BraveWrite #vss365 #haiku
#PoetryCommunity #adorn🌹
#tanka #RareDisease 🥀
#healthcare #HealthInsurance
#PassTheBill

youtu.be/nkqVm5aiC28?si…
“Health is a crown that the healthy wear, but only the sick can see it.” By Imam Shafi’ee
January 8, 2026 at 10:55 AM
drtimos.bsky.social
#Raredisease innovation accelerated in 2025, but access lagged. #Gene and #RNA therapies advanced rapidly as pricing, evidence standards, and delivery became the true constraints. Execution, not science, defined success. 👉 Read more: http://dlvr.it/TQDDXp #LucidQuest
Rare Disease 2025 Preview: From Orphan Innovation to a System-Level Stress Test - LucidQuest Ventures
Rare disease innovation surged in 2025 as gene and RNA therapies outpaced regulatory, access, and pricing systems.
dlvr.it
January 8, 2026 at 10:02 AM
kuglerelisabeth.bsky.social
Some of the most important conversations in science happen when we slow down and really listen.

Keep It Science Episode 5 explores rare 🫁 lung diseases alongside Prof Dr Allan Lawrie and Dr Sterghios A. Moschos. Hosted by Dr Nick Gaunt.

🔗 Listen: youtu.be/8CQN9U2h388
#RareDisease #scicomm
January 8, 2026 at 9:30 AM
erneurogen.bsky.social
Join @erneurogen.bsky.social & ERNs across Europe for a crucial webinar:

🛠️ Medical Device Shortages & Regulatory Constraints
🗓️ Wed 21 Jan @ 18:00 CET
🎙️ With clinicians, regulators & industry experts
🔗 Register: bit.ly/eUROGEN21Jan26

#EUfunded #HealthUnion #RareDisease #ERNs
January 8, 2026 at 9:14 AM
smanewstoday.bsky.social
Find adaptive clothing ideas that make SMA life more comfortable: https://bit.ly/45uFRZI

#SMA #SpinalMuscularAtrophy #SMANewsToday #Bionews #RareDisease
January 8, 2026 at 12:02 AM
drjudystone.bsky.social
Congratulations to @cureid.bsky.social @idepiheather.bsky.social (Heather Stone) and team for publication: CURE ID: A Platform to Collect Real-World Treatment Data for Drug Repurposing in Rare Genetic Disorders
onlinelibrary.wiley.com/doi/10.1002/...
#DrugRepurposing #RareDisease
onlinelibrary.wiley.com
January 7, 2026 at 11:26 PM
nowbreezing.ntw.app

Bluesky's Top 10 Trending Words (past 10min):
💨x74 - minneapolis
💨x73 - sesko
💨x1* - epstein 🔓
💨x63 - renee
💨x1* - democracy 🔓
💨x36 - frey
💨x1* - ukraine 🔓
💨x33 - semenyo
💨x31 - unarmed
💨x27 - self-defense
*🔓 = Unlocked Emergency Words (see img)

#FreePalestine 🇵🇸

(Something not right? Reply!)
Word Cloud; its top words (sorted by weighted frequency, descending): minneapolis, sesko, epstein*, renee, democracy*, frey, ukraine*, semenyo, unarmed, self-defense, murderer, gaza*, untrained, murdered, geraldo, dorgu, joelinton, gestapo, blooded, agent, noem, walz, snowballs, nicole, miley, fatally, windshield, murdering, ice, abolish, masked, minnesota, caissie, thugs, shoots, murder, moyes, kristi, dhs, rapunzel, officer, minnesotans, @governorwalz.mn.gov, šeško, cabrera, abolishing, thug, riots, ice's, croft, agents, terrorize, shooting, murderers, mn, live-action, homan, cold-blooded, agitator, victim's, vehicle, slush, observer, mobilize, manheim, grönland, grealish, feds, citizen, burnley, bournemouth, babbitt, antoine, amorim, teagan, streets, sherwood, ordeal, mpd, header, fulham, floyd, executed, enforcement, daylight, complying, 2026-01-07, valentine's, raytheon, pretext, peacefully, neige, marinera, intends, heatwave, fleeing, executing, everton, err, disorderly, cristian, bovino, barrels, arrest, 1-1, $500b, 木曜日, wwiii, wissa, wegner, viciously, troubled, trillion, tottenham, terrorists, terrorist, terrorism, squads, spurs, shooter, rutte, rittenhouse, remakes, recovering, reap, protestors, officers, motorist, martial, marlins, lethal, lafayette, keane, insurrectionists, hump, edgardo, domestic, cucurella, civilian, broad, ange, accessory, 2-2, 1﹐664, 1/8, $50﹐000, woman, witnesses, uprising, ubisoft, trained, suv, striker, shot, shoot, shaw, sanctioned, salto, rivera, rioters, resisting, relegation, reece, provoking, protests, protesters, prosecuted, prosecute, parish, paramilitary, owen, offshore, offenses, obstructing, obstruct, münkler, murders, murderous, milo, militias, mcneil, leeds, insurrection, innocent, identified, haaland, guimaraes, flynn, execution, escalate, emboldened, ember, dietary, deficit, deadly, cubs, crackdown, cpr, cops, chauvin Hashtag Cloud; its hashtagged words/phrases (sorted by weighted frequency, descending): nufc, ice, minneapolis, lufc, coys, mufc, minnesota, fuckice, abolishice, tweetchoir, thetraitors, newlee, ismuasmonthofmusic, humpday, fpl, avfc, ttrpg, politics, greenland, maga, burmun, thfc, shorts, safc, love, gpose, fucktrump, berlin, naturephotography, murder, iceshooting, frankout, female, evewol, coyg, boutot, wwfc, viera, tech, survivor, sport, snow, snl, reneenicolegood, premierleague, poem, originalart, nintendoswitch, new, netflix, media, marvel, landscape, interest, innovation, icemurder, human, fulche, florida, ffc, dlxp, digitalartist, dhs, deadlinewh, crochet, cpfc, commissions, comicbooks, bottom, artificialintelligence, arsenal, wethepeople, vwfc, undertale, ukbirding, tiktok, speirgorm, sketches, raredisease, puppy, noem, mha, lewdrp, jazz, iykyk, inflation, indivisible, indiepop, imnottakinganymore, icegestapo, ia, hiring, hackernews, gervtuber, feminization, everton, erotica, efc, eeuu, diy, classicmono, chatgpt, cfc, astrophysics, artistfave15, agdq2026, afcb, 942, 10, 초대, 변녀, 네토, 短歌, 東方project, 東方, 占い, 初音, 写真好きな人と繋がりたい, 共同通信ヘイト問題取材班の凍結を解除せよ, 企業, 人生, ミコッテ, ニュース, ガンプラ, うちの子, いまそら, êtamundomelhor, ynwa, writingprompt, writers, workfromhome, women, woman, wildfires, whitesupremacy, waterwednesday, warrenbuffett, waifu, vienna, vengadoresdoomsday, valorant, valentinesday, utwx, utv, uspolitics, uspoli, unionstrong, ukwildlife, udinese, tシャツ, tvnews, ttrpgcommunity, truth, trumps, trees, travelphotography, traitorsuk, toyphotography, tour, torino, tomorrowspaperstoday, today, tl, timwalz, teamlocked, team, teagancroft, tdick, tbs, tarot, tangled, tamron, synthpop, switch, supernatural, superman, sunrise, sundayhsr, streetart, strategy, stockmarket, stellarblade, steamdeck, staticmoth, starcitizen, spurs, sports, splatoon3, spicycontent, solodev, socks, smutsky, smoke, slave, ski, shortstory, shinypokemon, sfwart, sfw, series Emoji Cloud; its emojis (sorted by weighted frequency, descending): 🥦, ↗️, 🩺, 🧊, 🤞🏽, 📕, ↘️, 🪙, 🤬, 🤦🏻‍♂️, 🤘🏼, 😶, 😙, 📎, 💅🏻, 👥, 👐, 🐿, 🇨🇴, 🅰, ✍, ⚽, ⚰️, ⚫, ⚪, 🫵🏽, 🪵, 🪦, 🪕, 🩹, 🧶, 🧱, 🧣, 🧚‍♀️, 🦪, 🦞, 🦔, 🦑, 🦆, 🦁, 🦀, 🥕, 🥑, 🥅, 🤺, 🤷🏾, 🤷🏼‍♂️, 🤠, 🤞🏼, 🤝🏾, 🤌🏻, 🛰️, 🛡, 🛠️, 🛑, 🛐, 🚪, 🙏🏽, 🙏🏻, 🙇🏻, 😫, 😡, 😓, 🗨️, 🖕, 🕸️, 🕳, 🕊, 🔶, 🔮, 📦, 📋, 📊, 📅, 📁, 💻, 💳, 💢, 💁, 👶, 👩, 👟, 👏🏾, 👍🏾, 👇🏿, 👇🏼, 👇🏻, 👂, 🐲, 🐯, 🐠, 🐚, 🐙, 🐕, 🏴󠁧󠁢󠁷󠁬󠁳󠁿, 🏴󠁧󠁢󠁥󠁮󠁧󠁿, 🏫, 🏪, 🏢, 🏠, 🏛️, 🏁, 🎼, 🎪, 🍳, 🍖, 🍅, 🌐, 🇵🇹, 🇬🇱, 🇩🇰, 🇧🇷, 🆘, 🅰️, ⬇, ⬆, ➡, ✋, ✊, ✂️, ⚔️, ♂, ☎️, 3️⃣ Emoji Legend 💨x# | "Increased Trending Rate" for a Word (meaningful value; "💨x8 - burger" will mean "burger" is trending at 8 times its own normal rate for this time of day) 🔓 | "Unlocked" Ongoing Emergency Word (such words are not weighted down by their own previous usage; their *ongoing* increased usage--weeks, months, years--would otherwise move them down the list.) [PREVIOUSLY USED] ⚠️💨# | "Trend Strength Rating" ⚠️🌀💨 | "Trend Strength Rating" (see above) ⚠️🌀 CAT. # | [same; "CAT." = Category, like hurricane] 💬# | 10min Post Count 🆎# | 10min Word Count 🙂# | 10min Emoji Count
January 7, 2026 at 9:40 PM
mgnews.bsky.social
What early treatment shows: https://bit.ly/45z30uN

#MyastheniaGravis #GeneralizedMG #MGResearch #ClinicalTrials #AutoimmuneDisease #RareDisease #MyastheniaGravisNews #Bionews
Myasthenia Gravis News headline: "Follow-up study finds early rituximab treatment may improve long-term outcomes in generalized Myasthenia Gravis (gMG)."
January 7, 2026 at 9:35 PM
cfnewstoday.bsky.social
Packing for a hospital stay becomes its own kind of ritual.

#RareDisease
January 7, 2026 at 9:30 PM