Top Tweets for #Raredisorders
New podcast episode! Krissy Dilger talks with Drs. Grace Gombolay and Varun Kannan about their multidisciplinary clinic, biomarker research, and clinical trials. Listen now at https://t.co/wLzmHi3Nb9
#Neuroimmune #Podcast #RareDisorders #CommunitySupport #SRNA
.@ChildrensPhila’s Dr. Yi Xing et. al developed a low-cost long-read RNA sequencing platform that revealed disease-causing variants in 88 children with a range of #RareDisorders —including a patient whose condition went undiagnosed for a decade. Read more: https://t.co/suGQW1UPPC

The evolving methodology of basket- analog trial enabling phenotypebased approaches to accelerate treatment in DEEs.
https://t.co/StOOZvmGYl
#epilepsy #ILAE #epilepsia #baskettrial #masterprotocol #raredisorders @KetteValente

Reza Shojaei (@mrezashojaei) on Making a Measurable Difference in Patients’ Lives
@PlasmaProteins
https://t.co/SKOExoOybf
#BleedingDisorders #Health #Hematology #Hemostasis #HemostasisToday #ImmuneDeficiencies #Medicine #PDMPs #PlasmaDonation #PPTA #RareDisorders #TransfusionMedicine #MedEd #MedX
Kleine-Levin Syndrome
Ever heard of people sleeping 20+ hours a day… for days or even weeks?
The wild part? It disappears for months or years, then comes back like a mysterious sequel.
Guess the fairy-tale name of this sleep disorder!
#KleineLevinSyndrome #RareDisorders

Thrilled to share our latest #research #publication on #CDKL5 deficiency and #stemcell #diseasemodeling. This critical work took a tremendous team effort over the years! #autism #intellectualdisability #epilepsy #raredisorders #neurogenetics
https://t.co/zyKECYdb0W
Big Win Thanks to @RareDiseases: 1 Single Law Removes Barriers for Rare Disease Patients in Alaska
https://t.co/qZfkwhhlTC
#AccessibleCare #Health #Hematology #HemostasisToday #Medicine #NORD #RareDiseases #RareDisorders #SAIMCoalition #SB133 #TherapyBarriers #MedEd #MedX
Hopefully they can access the proper medical treatment #raredisorders #medical #genetics #doctorexplains
Unlock the mystery of Leontiasis Ossea 🦁, the intriguing "lion face syndrome" with facial and cranial bone overgrowth. Remember, it's a symptom of underlying conditions, not a standalone disease! #medicalmystery #raredisorders

Please share this with others who may be eligible — every story matters. #RareDisorders #Rarediseases @RareDiseasesIE @rarerarely @a_j_mcknight @RAiNAllIreland @HelenMcAneney @Melissa_Kinch_ @orladoyle_econ @NI_RDP @22Q11_Ireland @CARLEENWALGAL @raisingrareness @SBH_PAG

Caring for a child with a #RareDisease comes with #financial, #social, and #emotional challenges. We want to understand your experience to create positive change.
Share your voice!
🎥 Watch short video https://t.co/MiN6gKMOpt
👉Take part in the SURVEY https://t.co/Esg9subaDa
Honoured to present our research on rare disorder genetics at #LorneGenome2025! Grateful for the opportunity to share insights on VPS52 variants in brain development & connect with experts in genomics. Excited for future collaborations! #Genetics #RareDisorders #BrainResearch

📢 We're Hiring – Engagement & Communications Officer! 🌟
To apply and find out more click here: https://t.co/dIY8do5imm
Join us in making a difference!
#Hiring #CommunicationsOfficer #CharityJobs #RareDisorders

New Brunswick has signed a $32-million agreement with the federal government to improve access to four drugs for rare diseases -- three for rare cancers and one for a rare kidney disease. https://t.co/case4xw8Co via @globeandmail @raredisorders #RareDisorders
Revive Therapeutics Provides Update on Research Study Evaluating Bucillamine for Nerve Agent Exposure
https://t.co/ksEi1hhv07
$RVV $RVVTF #Bucillamine #InfectiousDiseases #MedicalCountermeasures #RareDisorders #NerveAgent #DRDC #DefenceRDC #FDA #HealthCanada

He diagnosed his rare disease using Google. Now he hopes artificial intelligence can do the same for others. @CHEO using #AI to help diagnose #RareDisorders, by @phildrost https://t.co/9r6aBQbDFy via @cbcwhitecoat @NightShiftMD
🌍✨ International Day of Persons with Disabilities ✨🌍
IDPWD is a day to celebrate the achievements and contributions of persons with disabilities as well as increase public awareness, understanding and acceptance.
#IDPD #InclusionMatters #DisabilityAwareness #RareDisorders

Ottawa boy with rare disease gets tickets to Taylor Swift show thanks to generosity of others https://t.co/mDzEEz3etB via @cbcnews #RareDisorders #SPG4
🌎 Opening Remarks by MEP Billy Kelleher
Early detection can transform lives. We need to ensure Member States have the right information to take informed decisions on which treatable #raredisorders should be included in their newborn screening panels.
#NewbornScreening #Screen4Rare

Revive Therapeutics Provides Update on Research Study Evaluating Bucillamine for Nerve Agent Exposure
https://t.co/HYLrE1iYO1
$RVV $RVVTF #Bucillamine #InfectiousDiseases #MedicalCountermeasures #RareDisorders #NerveAgent #DRDC #DefenceRDC #FDA #HealthCanada

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