@nordrare.bsky.socialNew #HR1 work requirements for Medicaid could cause eligible people with #RareDiseases to lose their health insurance.
NORD created model policy language for state #Medicaid agencies to prevent unintended coverage loss. Read and share it with your state officials: rarediseases.org/medicaid-wor...
@erdera.bsky.social👀 What’s on at the #REACTCongress 2027?
The programme and speakers are waiting to be explored! 🧬🎤
Discover what’s on the agenda, who’s taking the stage and start planning your Congress experience. 👉 https://loom.ly/_fGiC04
@beesmartypants.bsky.social#disability #RareDiseases #autoinflammatory
Crowd Sourcing for leads to a diagnosis after yet, Another, trip to ED
I’m fluctuating between hyperthermia & hypothermia multiple times a day Temp range 93.6 -99F during “fever”, 96.6 normal body temp. Rheumatologist is like 🤷♀️
Neuro & PCP same
Help plz
@beesmartypants.bsky.socialAnybody else get the letter from Silver Scripts telling us we have to pay more for another drug supplemental insurance in 2027?
The timing couldn’t be more perfect. A punch to the face reminder
VOTING BLUE SAVES LIVES
@beesmartypants.bsky.social#RareDiseases #FMF #IIH #HidradenitisSuperativa #RA
Without access to AFFORDABLE healthcare too many of us suffer for decades from PreVentAble pain & loss of functions
Too many of us relinquish good health for other essentials
It’s past time for
#MedicareForAll
Vote blue to save lives
@ataxiacentreucl.bsky.socialIt's good to see more coverage about #FriedreichsAtaxia and the need to facilitate access to #treatment Skyclarys (Omaveloxolone) already approved
#AccessOmvavNow #RareDiseases
you can read more here: www.ataxia.org.uk/accessomavnow/
www.belfastlive.co.uk/news/tv/nort...
@dhsc-answers.bsky.socialA question on #RareDiseases #HealthServices tabled by Jim Shannon on 15-09-2026 has been answered by Mr James Frith. https://questions-statements.parliament.uk/written-questions/detail/2026-09-15/29917
@drtimos.bsky.social🚀 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/TVt82T #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT
@erdera.bsky.social🧬 What ethical challenges arise in ATMP research, and how can resolving them become an advantage?
Join the next #ERDERA Ethics & Regulatory Webinar with Joana Namorado.
📅 2 Nov 2026 | 13:00 CET
💻 Online
🔗 Find out more & register: https://loom.ly/lZ8lmm8
#RareDiseases #ATMP #ResearchEthics
@bffo.bsky.socialFrom #OUP 's #DATABASE journal | Ensemble multi-retrieval methodologies with reasoning model decision in multi-step biomedical QA | #OpenScience #BioCuration #LLM #QA #QuestionAnswering #Wikipedia #RareDiseases | 🧬🖥️🧪🔓🇰🇷
⬇️
academic.oup.com/database/art...
@iic-umu-imib.bsky.social🧠🐟 Excited to share our new study on NEXMIF-related encephalopathy!
We developed a zebrafish model, identified novel modifiers of hyperactivity (ENTPD3, DDX43 & KBTBD7), and established a platform for drug discovery.
@hlm4rare.bsky.social"Patients have to be there at the table to co-decide with the other stakeholders." 🤝
Our take-home message: every time there is a new mechanism or a new governance, patients have to be at the table.
@hlm4rare.bsky.social#RareDiseases are "an opportunity for innovation", not a cost. 📊
For the ERNs, cross-border registries are "not a choice", but a methodological requirement: there are not enough patients in one country.
A common ethical framework? "Yes, we need it."
@hlm4rare.bsky.social"Access starts with a proper diagnosis."
The way forward: faster decisions on what goes into newborn screening panels, more discussion on newborn screening, and a rare disease action plan, key for both diagnosis and other therapies.
👉 hlm4rare.eu
@hlm4rare.bsky.social"Europe does not lack clinical expertise." 🔬
Holm Graessner's message: we can do this now. We have what we need in place. What we need is trust, and the resources to deliver.
👉 Follow our work: hlm4rare.eu
@hlm4rare.bsky.social"If not now, when?" Maurizio Scarpa's answer: it's now. ⏳
Because a child born in Vilnius, Valencia or Vienna should have the same chance, not one that depends on their postcode.
👉 Follow our work: hlm4rare.eu
@nmd4c.caThe NMD4C October 2026 Newsletter is now live, featuring the latest updates from across Canada’s neuromuscular community.
🔗 Read this month’s edition: zc.vg/y4zCw?m=0
@drtimos.bsky.social🧬 Gene & Cell Therapy | #RareDiseases 🚀
#LucidQuest delivers sharp insights to help biotechs, investors & BD teams stay ahead in fast-moving markets.
📩 [email protected] or http://dlvr.it/TVqbST
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
@mitoworld.bsky.social#Mitochondrialdiseases are central in the #rarediseases research realm, but recently are getting more recognition for the #metabolicdysfunction associated. @natmetabolism.nature.com looks at the metabolism angle in this latest viewpoint:
@livinglfs.bsky.social