Novel Medicines For Patients With Rare Diseases - Rich Daly - President and CEO, #CatalystPharmaceuticals Inc., joins me on Progress, Potential, And Possibilities #LambertEatonMyasthenicSyndrome #DuchenneMuscularDystrophy #Epilepsy #RareDiseases #OrphanDiseases www.youtube.com/watch?v=UrBV...

Episode 3 of Dr. Bonsib's latest lecture series on cystic kidney diseases is here! This episode is part 2 of the discussion on congenital anomalies of the kidney & urinary tract. 

Feel free to send in any questions! #renal #pathology #orphandiseases

#ICYMI we moderated a panel at the recent #WorldOrphanDrugCongress to discuss “The Future of #DrugDevelopment in #Rare and #OrphanDiseases: Navigating #Regulatory Challenges and #ClinicalInnovation." Catch the key takeaways at buff.ly/jXRnRn5

#RareDiseases #FDA

The Unmaking of American Medicine No new NIH grants have been awarded. Graduate programs are curtailing admissions. An entire generation of physician scientists are on the line as the system begins to unravel. What's really at stake?

meilanhan.substack.com/p/the-unmaki...

The disturbing impacts of cutting grant funding

#Research #NIH #MSTP #Orphandiseases #Disease #Healthcare #R&D #DOGE #Trump #MAGA #Science #STEM #Medical

End of day one, after an inspiring session on “Empowering #data” with great speakers
* Nicole Vasilevsky, US 
* Sabine Österle, CH 
* Thomas Hellebrand, BE 
* Marieke Bak, NL 

#RAREvolution
@erdera.bsky.social #IRDiRC #REACTCongress2025 #RareDiseases #OrphanDiseases

@

Illuminating session this morning on “Diagnosing #rarediseases: from NBS to machine learning” with outstanding speakers
#collaborationiskey
#RAREvolution!
@erdera.bsky.social #IRDiRC #REACTCongress2025 #OrphanDiseases
@vyepez.bsky.social

This is a start! 🤘🏼

#RAREvolution!
@erdera.bsky.social #IRDiRC #REACTCongress2025 #RareDiseases #OrphanDiseases

D-day #RAREvolution!
@erdera.bsky.social #IRDiRC #REACTCongress2025 #RareDiseases #OrphanDiseases

Tomorrow will start the 8th edition of the RE(ACT) Congress! We are ready, and you? It’s time for #RAREvolution!
@erdera.bsky.social #IRDiRC #REACTCongress2025 #RareDiseases #OrphanDiseases

Original post on wisskomm.social

Zum #TagDerSeltenenErkrankungen möchten wir noch mal auf eine Publikation vom Januar hinweisen:

Orphan Drugs: Stärkere Orientierung am medizinischen Bedarf notwendig

Ein Team aus dem IQWiG hat Nutzenbewertungen von Orphan Drugs aus gut 10 Jahren analysiert.

Onkologische Indikationen sind […]

Orphan Drugs: Stärkere Orientierung am medizinischen Bedarf notwendig Ein Team aus dem IQWiG hat Nutzenbewertungen von Orphan Drugs aus gut 10 Jahren analysiert. Onkologische Indikationen sind überrepräsentiert, obwohl hier in den meisten Fällen bereits aktive Behandlun...

Zum #TagDerSeltenenErkrankungen möchten wir auf eine Publikation vom Januar hinweisen:

Orphan Drugs: Stärkere Orientierung am medizinischen Bedarf notwendig

Ein Team aus dem IQWiG hat Nutzenbewertungen von Orphan Drugs aus gut 10 Jahren analysiert.
www.iqwig.de/presse/press...

#OrphanDiseases

@mitchmcconell.bsky.social
Thank You Senatorsaurus 🦖!
#MitchMcConnell was the ONLY Republican to oppose this nonsense with stupid Bobby. It’s very little, too late, unfortunately for those of us with #idiopathic #ChronicIllness, #OrphanDiseases, & #geneticconditions. Also huge loss for #Humanity

Full Program - React-congress

👉 Register today and check out the complete program: loom.ly/npx4DUQ

#RAREvolution #REACTCongress2025 #RareDiseases #OrphanDiseases
@erdera.bsky.social #IRDiRC

🌟 Only 9 days remain to register for the RE(ACT) Congress & IRDiRC Conference 2025, the one-of-a-kind international Congress on #RareDiseases research.

✨ Join the #RAREvolution
#REACTCongress2025 #RareDiseases #OrphanDiseases
#IRDiRC #ERDERA @erdera.bsky.social

📅 Register here: loom.ly/FOgBwLw

We are pleased to announce that the RE(ACT) Congress and IRDiRC Conference 2025 have extended the regular registration and abstract submission deadline until the end of January!🎉

👉 Register: loom.ly/FOgBwLw

#RAREvolution #RareDiseases #OrphanDiseases #IRDiRC #ERDERA
@rarediseasesint.bsky.social