📢 Call for Papers
I’m pleased to be Guest Editor for a Special Issue in Cells on:
Cellular & Molecular Mechanisms Behind Liver Inherited Metabolic Diseases and Novel Therapeutic Strategies
Original research & reviews welcome.

📅 14 May 2027
#RareDisease #Metabolism

MaveMD: a functional data resource for genomic medicine #RareDisease #Genetics link.springer.com/article/10.1...

#EUfunded #RareDisease #ERNs #PedUro #SoMe4PedSurg @ec.europa.eu @jardinjointaction.bsky.social @espu.bsky.social

This is part of our #VWD100 campaign, marking 100 years of #VonWillebrandDisease throughout 2026. Each month, we shine a light on a different aspect of #VWD.
#beVWDaware #VWDunited #VWDtogether #BleedingDisorders #RareDisease

Our team will be on the ground at #ASN2026 in Denver analyzing the latest data releases, strategic shifts, and clinical breakthroughs.

Detail about CVrg Conference Coverage: cv-rg.com/conferences/

#ASN #KidneyWeek #Nephrology #PharmaIntelligence #MarketAccess #CKD #RareDisease #Biotech

Stay in the know—and never miss an update! https://bit.ly/3FHecMk

Sign up for the weekly newsletter, because knowledge is power, and you’re not alone!

#Angioedema #HAE #PatientSupport #Newsletter #RareDisease #AngioedemaNews #Bionews

A cross-tissue splicing signature as a quantitative biomarker for ReNU syndrome #RareDisease #Genetics www.medrxiv.org/content/10.6...

Take a moment today to recognize your progress: https://bit.ly/4kUuC33

It’s easy to get caught up in “what ifs” about the future, but don’t forget to celebrate how far you’ve come.

#Angioedema #HAE #ChronicIllness #CaregiverLife #ProgressNotPerfection #RareDisease #AngioedemaNews #Bionews

Take a closer look: https://bit.ly/4AFrb8M

At MDA Engage, a neurologist outlines advancing myasthenia gravis treatments, including CAR T-cell therapy, and daily energy management tips.

#MyastheniaGravis #RareDisease #MG #MyastheniaGravisNews #Bionews

#MetabolicHealth #GeneTherapy #RareDisease #HealthLongevitySecrets

Full breakdown coming on the Health Longevity Secrets podcast www.youtube.com/@RobertLufkinMD (4/4)

Balamuthia Infection is a rare, often fatal brain infection caused by a free-living ameba in soil, dust, and water. Early signs can include slow-healing skin wounds, fever, and headache.

Learn more: https://dub.sh/BALA

You may be rare, but you're not alone!
#rareDisease #rareDiseaseAwareness

Intellectual deficit, X-linked - psychosis - macroorchidism https://gamuts.net/x/34680 #RareDisease #MedEd #FOAMrad

Drop a ⏳ if your symptoms change hour to hour. #RareDisease

Garetosmab (Pasatru) sharply reduces new bone lesions in adults with FOP, phase 3 Lancet trial shows; FDA approved Aug. 19, 2026

#fop #garetosmab #raredisease #fda

I am excited to be an Exhibitor at Rare New England's 2026 Annual Conference, Advancing Rate Together! 💙 Turning Rare Disease Experience Into Advocacy

Learn more: https://www.rarenewengland.org/

You may be rare, but you're not alone!
#rareDisease #rareDiseaseAwareness

Meet the 2026 NORD #RareImpactAwards honorees! 🎉

From breakthrough treatments to research, policy change, medical education, and community advocacy, this year’s honorees are moving the #RareDisease field forward.

Read about each of the honorees: bit.ly/4hrahn3

A single treatment that could end HAE attacks for good: https://bit.ly/3FHecMk

Gene therapy research is bringing us closer to that reality.

#HAE #HereditaryAngioedema #GeneTherapy #CRISPR #RareDisease #AngioedemaNews #Bionews

#urologyweek #VHL #RareDisease @uroweb.org @ec.europa.eu @jardinjointaction.bsky.social @erknet.bsky.social

#EUfunded #RareDisease #ERNs #PedUro #SoMe4PedSurg @ec.europa.eu @jardinjointaction.bsky.social @espu.bsky.social

Discover more: https://bit.ly/4xOMWjW

For columnist Shawna Barnes, myasthenia gravis (MG) disease progression is like waking up every day in a body that feels less like your own.

#MyastheniaGravis #RareDisease #MG #MyastheniaGravisNews #Bionews