Hemophilia Therapy Trends 2026: Market & Innovation Explore the latest hemophilia treatment trends, including adoption of gene therapy and non-factor therapies, key challenges, and US vs EU market insights.

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

Why India's general doctors need urgent rare disease training | Research Matters A new study reveals that while patient groups and specialists fight hard for rare disease awareness in India, a critical lack of knowledge among general doctors is leaving millions without care.

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 Rare Diseases 2026 - Vydya Health - Find Providers, Products. The 5th MENA Congress for Rare Diseases 2026 will take place from 3 to 5 September 2026 at Beach Rotana Hotel, Abu Dhabi, United Arab Emirates.

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

22-23 PHARMA - Fast and the curious This month: the NHS as a dystopian game of Tetris, the GLP-1 explosion and its impact on future meds, plus deep dives into AI in pharma, reproductive health, and Trump’s healthcare influence. Also, co...

#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.

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 | The-14 Discover four lesser-known types of dementia, their symptoms, and why early recognition matters for better care and support beyond memory loss worldwide today!

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

Travelling with SBB: Dealing with invisible disabilities on public transport and fostering greater understanding through small gestures – Swiss Angiodysplasia Association

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

The silent majority: RNAs that don’t make proteins Once considered cellular junk, non-coding RNAs are emerging as key players in everything from brain development to cancer — with much still to be discovered

"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 and Rare Disease Intelligence - LucidQuest Strategic insights for gene and cell therapy from R&D through commercialization. Primary KOL research, competitive analysis, and investment diligence in rare diseases.

🧬 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

Diagnosis and treatment of rare genetic diseases Rare genetic diseases affect approximately 3.5 million people in the UK. How can genetic technologies, policies and initiatives in healthcare support diagnosis and treatment?

New POSTnote briefing on 'Diagnosis and treatment of
rare genetic diseases' (POSTnote 763, doi.org/10.58248/PN763) has been published, with contributions from the UOI's Dominic Wilkinson.

@oxpolicyengaged.bsky.social
#PhilosophyMatters #ethics #postnote #rarediseases

📧✨ The fourth edition of the JARDIN newsletter is coming soon!

Subscrite today and be among the first to discover:
🌍 Updates from work packages
💼 Highlights from Member States
📅 ERN news
👥 Stories and developments impacting people living with #RareDiseases

👉 https://jardin-ern.eu/newsletter

📍 Yesterday the RealiseD Playbook team met yesterday at the European Medicines Agency to exchange ideas on making the project’s Playbook relevant and useful. Early discussions like these help ensure different perspectives are considered from the start. 🤝 #RealiseD #ClinicalTrials #RareDiseases

📍Last week, ERDERA joined the TETECOU Research & Innovation Day.
🎤 ERDERA Project Manager Başak Uysal highlighted how coordination, data sharing & cross‑stakeholder collaboration can accelerate rare disease research across Europe.
#RareDiseases

A promotional share-pic for the conference. Below the Title there’s a scenic view from Warsaw and the date 09.-10. April and a QR code pointing to the conference program at https://ern-ithaca.eu/events-news/eurondd/program/program-2026/

I’ll attend the #EuroNDD Workshop in Warsaw in April. Anyone around here going there as well? —> let’s connect!

I’m looking forward to present our work at www.findme2care.de AND hear about all the other projects.

#humangenetics #genetics #RareDiseases #RareDisease #ERN #ERNIthaca #patientregistry

Swiss VASC-RN: An important step – and a major challenge – Swiss Angiodysplasia Association

🇨🇭 Switzerland just recognised 42 reference centres for rare diseases, incl. a vascular network (Swiss VASC-RN). Great start – but networks only work if they truly bundle cases & expertise, not just share information. More on our website: angiodysplasie.ch/en/swiss-vas...
#RareDiseases #SwissVASCRN

🌍 European Reference Networks (#ERNs) connect rare disease experts across Europe. Within #ERDERA, ERNs help bring clinicians, researchers & patients together to share knowledge, advance research and improve care where it’s needed most. Expertise, connected. 💙 #RareDiseases

🧬A few days ago, CNAG participated in the 4th #Screen4Care consortium meeting, an opportunity to explore digital omics and reflect on advances in rare disease diagnosis through genetic newborn screening and digital technologies

#RareDiseases #Research #Horizon2020 #CNAG
@ucddublin.bsky.social