Our first experiment, nearly a decade ago, was not the result we hoped for. But in hindsight, it gave us a valuable lesson on matching models to experiments. Paper on our findings: biorxiv.org/content/10.110… Blog post - what we learned & why now: cureffi.org/2022/10/12/les…

To map the landscape of neurodegenerative disease clinical trials we analyzed 20 years of trial registrations. The paper: nature.com/articles/s4159… My blog post: cureffi.org/2022/10/24/pre… Key findings in this thread 👇

We are honored to tell our story in this episode of NPR's Short Wave from Gabriel Spitzer & Emily Kwong npr.org/2022/11/14/113… - part of a fantastic series they are doing on prions. 2nd half to come next week!

In Part 3 of NPR's Short Wave's series on prions, we describe our scientific strategy and the road to a cure npr.org/2022/11/14/113… — thanks Gabriel Spitzer and Berly McCoy, PhD for working with us to tell our story!

Our end-of-year newsletter: prionalliance.org/2022/11/29/end… "We must be constantly vigilant, vocal, and feisty; we must be, to a judicious degree and at the right moments, trouble."

If an ASO lowers target RNA 50% in bulk brain tissue, is it 50% in all cells or 100% in half? Are cell types all impacted equally? Does CSF reflect neuronal target engagement? We did single-cell seq in ASO-treated mice & NHPs to find out doi.org/10.1101/2023.0… Thread 👇

ASO activity at single cell resolution - now published as a Nucleic Acids Res Breakthrough article: academic.oup.com/nar/advance-ar… Blog post on implications for prion prevention: cureffi.org/2023/05/16/aso… Tweetstorm: x.com/cureffi/status… Thank you @OnoPharmaFound for funding this 🙏

Prion2023: registrations are open at prion2023.org #Prion2023 PRION2023 AD/PD - Advances in Science & Therapy Universidade Algarve MBExC

Happy June! Here is our mid-year newsletter prionalliance.org/2023/06/01/mid… touching on the scientific progress from all corners that keeps us optimistic.

Congratulations to Sonia M. Vallabh, Ph.D., and Erik Minkel, Ph.D., the winners of the OTS Paper of the Year Award - Basic Research Category! #ots23 #ots #oligomeeting #oligotherapeutics #biotech #biotechnology #science #research #barcelona Eric Vallabh Minikel

I first got to know Sonia Vallabh last year and am honored to call her a friend today. Her story has taught me so much about how to live with extreme uncertainty and still find equanimity. So thankful she’s sharing it with us all today. ❤️: apple.co/46i98VS

As promised, here is a new blog post unpacking what we know so far about the design of Ionis's upcoming PrProfile trial of ION717 in prion disease: cureffi.org/2023/12/04/fir…

Sonia with our semiannual newsletter. Human trials are finally around the corner. The outcome could be anything. It is the long view that gives us hope. A good time to remember the Stockdale Paradox. prionalliance.org/2023/12/05/end…

We've just updated our FAQ, which for years has said there are no ongoing trials in prion disease, to reflect that the first trial of a PrP-lowering ASO is now recruiting prionalliance.org/faq/ Trial posting: clinicaltrials.gov/study/NCT06153… Ionis statement: prionalliance.org/media/2023/10/…

Beautiful, inspiring journalism by Meredith Wadman science.org/content/articl… -- the Hoffmans of Reed City, MI are our new heroes and we are so happy for their ray of sunshine!

We long assumed increases in NfL always meant neuronal damage, and decreases in NfL meant amelioration thereof. The data we report today have now made us think there are exceptions. medRxiv: medrxiv.org/content/10.110… Blog post: cureffi.org/2024/05/06/wha… 🧵👇

Our mid-year newsletter. The ASO is in the clinic and patients are really showing up. Sonia's TED talk, released today, pleads the case for prevention. Our most ambitious donation matching campaign yet. Let's do this! prionalliance.org/2024/05/30/mid…

Sonia with our end-of-year update on developing a therapy for prion disease. Two new efforts from our lab: CHARM and divalent siRNA, and an update on the Ionis clinical trial. Support our work with a 1:1 matched donation through end of 2024. prionalliance.org/2024/12/02/whe…

Today we introduce a new drug candidate for prion disease: a PrP-lowering divalent siRNA. A 4-year collaboration with RNA Therapeutics Institute enabled by NINDSfunding IGNITE & URGenT mechanisms. biorxiv.org/content/10.110… Our hope is to advance this to the clinic, wish us luck!

Here is Sonia with our semi-annual newsletter prionalliance.org/2025/06/09/all… Donations will be matched 1:1 through end of June!