Latest posts tagged with #Rarediseases on Bluesky
🙌 Outstanding conference by @ewanbirney.bsky.social! Yesterday, we celebrated 25 years of the Human Genome Project with one of its co-authors, who shared first-hand insights into its impact on biomedicine, applications in #RareDiseases & #cancer, and the future of AI
With @easigen-eu.bsky.social
💡 New ERDERA Knowledge Pill! What are ATMPs and how are they changing how we think about treatment, especially for people living with rare diseases?
🔎 Learn more: https://loom.ly/pjOwax0
#ATMPs #AdvancedTherapies #RareDiseases #ERDERA
Sam chose to #Go4Garvan to support our scientists’ vital work in identifying, understanding, and managing rare diseases. We can’t wait to cheer you on, Sam!
Learn more or support:
https://ow.ly/rqgA50YGjzl
#City2Surf #MedicalResearch #RareDiseases #SupportScience #SydneyRun
How is Bulgaria advancing rare disease policy in line with Europe?
ERDERA speaks with Prof. Radka Kaneva and Dr Petia Stratieva of the National Mirror Group. 👉 Read the full interview: https://loom.ly/Cv6fEqU
#RareDiseases #ERDERA
Amicus Therapeutics Hits $14.46 52-Week High: Amicus Therapeutics reached a 52-week high of $14.46 on Apr 7, 2026 (Investing.com), signaling renewed investor interest in its rare-disease franchise and… 👈 Read full analysis #AmicusTherapeutics #StockMarket #Investing #Healthcare #RareDiseases
ERDERA Grant Application has been submitted today for NEWCOMS CONNECT project. Congratulations to @fundaciondravet.bsky.social for all the effort done preparing the consortia and making the proposal so exciting for a more healthy society. Big Thanks! #rarediseases #coa #neurosciences #outcomes
🇵🇰 Proud moment!
Muscular Dystrophy Pakistan (MDP) joins RDI as an Associate Member 🌍
A big step for rare disease advocacy in Pakistan—strengthening global collaboration, patient support & access to care.
#RareDiseases #MDP #RDI #Pakistan
🧬 Gene & Cell Therapy | #RareDiseases 🚀
#LucidQuest delivers sharp insights to help biotechs, investors & BD teams stay ahead in fast-moving markets.
📩 info@lqventures.com or http://dlvr.it/TRttVM
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
What’s shaping the future of hemophilia treatment?
Innovation, cost, and patient adherence are key drivers.
Get the full insights in our latest case study 👇
www.sperresearch.com/Case-Studies...
#HemophiliaTreatment #GeneTherapy #HealthcareInnovation #PharmaTrends #BiotechIndustry #RareDiseases
New study exposes India’s healthcare knowledge gap
To read more:
researchmatters.in/news/why-ind...
#rarediseases #diagnosis #medicalfacilities #lifesavingtreatment #scicomm #science #sciencecommunication #IndianScience #ScienceinIndia
5th MENA Congress for Rare Diseases 2026
3-5 September 2026
United Arab Emirates
www.vydya.com/events/5th-m...
#RareDiseases #CellTherapy #RareBoneDisorders #RareImmuneDiseases
#MedicalConference
When Celine Dion shared that she had Stiff Person Syndrome, we had to look it up.
We know its very rare, but if you live with SPS or know someone who does, please share something you wish people knew 🩵🦓🩵
#stiffpersonsyndrome #raredisease #chronicillness #rarediseases #SPS
5.1 million people across Europe are living with rare cancers, highlighted by a pink map of European countries.
#RareDiseases & #RareCancers are not separate worlds - patients face similar challenges, from diagnosis to access to care.
Through the Rare Cancer Advocates Network, EURORDIS is working to improve outcomes and ensure no patient is left behind. 👉 Read more: https://go.eurordis.org/rarecancers
#HealthCommunication #HealthPromotion #HealthEducation #GlobalHealth #SpecialSections #LongCovid #RareDiseases
@tandfresearch.bsky.social @tandfonline.bsky.social
A graphic announcing that the CIRM Board approves $111M for discovery and clinical research. It details $80 million for six new discovery grants and $31 million for three clinical trial grants, aimed at developing treatments for both rare and common diseases. The CIRM logo is visible at the bottom right.
CIRM's governing board approved over $111 million to support advances in discovery and clinical research for children's #RareDiseases, #VisionLoss, and treatments for #neurodegenerative conditions. Learn more: bit.ly/4dtnLwY
Rarity doesn’t just define the disease, it shapes the experience: isolation, invisibility, and the burden of becoming an expert to navigate care.
📢 @thelancet.com editorial calls for a profound shift in how we use language in #rarediseases. Words are part of care! @rarediseasesint.bsky.social
It's been 2 years since Cure MFM13 (previously Cure HSPB8) was founded by Todd King!
Since then our main goal did not changes. We are working together to find a treatment and a cure using state-of-the-art technologies and advances in science.
#MFM13 #2anniversary #RareDiseases
#mRNAvaccines #GLP1 #obesity #CARTcelltherapies #oncology #PCSK9inhibitors #HepatitisC #Immunotherapy #checkpointinhibitors #RSVvaccines #Genetherapies #rarediseases #antivirals #Biosimilars #expandedaccess #biologics #HIV #Migraine #CGRPinhibitors #antifungals #precisionmedicine
zurl.co/3UDz7
Rare Disease Group Urges FDA Clarity: Advocacy group urged FDA clarity on Apr 1, 2026; ~7,000 rare diseases affect 25–30M Americans, raising concerns about development timelines and investment risk. 👈 Read full analysis #RareDiseases #FDA #HealthAdvocacy #MedicalResearch #PatientRights
🔬 How do we improve diagnosis for people with undiagnosed conditions?
📅 30 April | #UndiagnosedDay 2026
🌟 Expert & lived‑experience panellists
👉 View the agenda and register: https://loom.ly/DbXzw0E
#RareDiseases #Diagnosis #HealthPolicy
We recently wrapped up @missionmsa.bsky.social 2nd Annual Capitol Hill Day. We more than doubled last years attendance and met with more offices than ever before. Thank you to our incredible advocates who made this possible!
#MultipleSystemAtrophy
#MSA #MSAawarenessMonth #RareDiseases
🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TRpD9k #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT
The four types of dementia most people don’t know exist
#Dementia #HealthAwareness #BrainHealth #Alzheimers #Neurology #MedicalEducation #PublicHealth #RareDiseases #MentalHealth #Healthcare #Awareness #Science #Caregiving #Aging
the-14.com/the-four-typ...
Great discussions at the #PICKED webseminars 👏
9 DC presentations, strong supervisor feedback, and real cross-project exchange.
More: www.projectpicked.eu/news-and-eve...
Next up: Tallinn (15–16 Sept)
#HorizonEU #MSCA #RareDiseases
Committed since 1998, #AMMi has been a founding member of IMP since 2011.
At @euromit-2026.bsky.social , we are joining forces for the 6,500 patients in France. Turning research into treatments: this has been our fight for nearly 30 years !
#euromit2026 #AMMI #IMP #Mito #RareDiseases
📢 New report released!
The IPOPI Central-Eastern European workshop brought together experts and patient representatives from 9 countries to strengthen awareness and advocacy for people living with PIDs.
🔗 Read the report here: ipopi.org/our-work/nmo...
#PID #RareDiseases #PatientAdvocacy #IPOPI
At all SBB travel centres, you can now pick up a lanyard with a card for “hidden disabilities”. The Hidden Disabilities initiative was founded in Great Britain, is active internationally, and SBB is also taking part.
angiodysplasie.ch/en/travellin...
#sbb #HiddenDisabilities #RareDiseases
"Non-coding RNAs turn out to regulate everything from embryonic development to immune responses to brain function. They help determine which genes get turned on and off, and when. They can promote cancer or suppress it."
This info is very promising for #Genetics / #RareDiseases + #ChronicIllnesses ⚕️
🧬 Gene & Cell Therapy | #RareDiseases 🚀
#LucidQuest delivers sharp insights to help biotechs, investors & BD teams stay ahead in fast-moving markets.
📩 info@lqventures.com or http://dlvr.it/TRlGZt
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
Six out of ten people find managing care challenging, shown with a young man lying down and a woman busy in the kitchen.
For many people with #RareDiseases, care coordination isn’t provided - it’s expected.
📊 65% navigate multiple services
67% say providers communicate poorly
7 in 10 spend significant time organising care
Integrated care is essential. 👉 Read more: https://go.eurordis.org/integratedcare
