Video Presentation from 11th Annual National CJD Conference

By CJDSGN29/05/2020News, Presentations

We now have permission to provide this video produced by Eric Minikel and Sonia Vallabh about Antisense Oligonucleotide (ASO) Strategy to reduce PrP that was originally shown at our 2018 Annual National CJD Conference in Melbourne, Australia.

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CJD Support Group Network

17 hours ago

Eric Vallabh MinikelEric Vallabh MinikelEric Vallabh Minikel• 1st• 1stPrion scientist at Broad InstitutePrion scientist at Broad Institute6d • 6 days agoPublished today, our reasons for optimism that success rates in drug discovery can be improved by picking the right targets using human genetics: lnkd.in/d7rUpnq8The lightning summary:- Drug programs supported by human genetics are more successful, almost regardless of the nature of the genetic evidence- Increase in success rate may be especially large for disease-modifying therapies- Genetics still underutilized in picking targetsHere is a thread breaking down the findings panel by panel: lnkd.in/d-4f5FGuIt was my privilege to work with the best on this project, and I want to say a huge thanks to Matthew Nelson for teaching me so much about complex trait genetics that I "should" know as a Broadie but didn't, and to Coco Dong for teaching me to write better code (finally, after all these years, joining the tidyverse) ... See MoreSee Less

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CJD Support Group Network

2 days ago

Congratulations to our colleagues and friends who make the Australian National CJD Registry (ANCJDR) one of the top prion disease surveillance units in the world.https://www.facebook.com/photo?fbid=830712455768198&set=a.560287242810722 ... See MoreSee Less

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CJD Support Group Network

2 weeks ago

Blog from Eric Minikel discussing the reasons for the clinical trial pause and what it means. ... See MoreSee Less

Why the ION717 trial is paused

www.cureffi.org

Probably because they filled the lowest dose group and need to analyze the safety and pharmacology data before escalating to a higher dose.

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CJD Support Group Network

2 weeks ago

The Ionis Community Statement explaining the pause for Phase 1 Recruitment in current clinical trials will be available on our website . They explain well the reasons for the pause. There are also FAQ's on page 2. Sites will continue to open including Australia but recruitment of new patients will only begin again when we are advised that the pause is completed. Patients who have already been recruited in sites already open globally will continue on the trial. Please read the statement for more details www.cjdsupport.org.au/trial-update-for-prion-diseases/ ... See MoreSee Less

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CJD Support Group Network

3 weeks ago

A call from Ionis this morning our time advised that there is now a pause in the recruitment for the clinical trial. Unfortunately The Australian site has not opened up yet so had not began recruiting. Opening up of sites will continue and once the pause is over I understand our site should be able to begin recruiting. The recruiting process has been faster than expected which has led to the need for a pause. We are waiting to receive a community statement so we can share more details with you. www.cjdsupport.org.au/trial-update-for-prion-diseases/April 6th, 2024We have been advised that Ionis has paused recruiting for their ION717 Human Clinical Trial for Prion Disease (CJD, GSS, FFI).We want to be clear that the study continues – only recruiting of new patients is paused at this time. We have not been advised at this stage when recruiting will commence again.Please understand that this is not uncommon in research. We will provide the Ionis Community Statement explaining the pause once it becomes available ... See MoreSee Less

Good News – Clinical Trial Update for Prion Diseases, Mar 2024 – CJD Support Group Network Australia

www.cjdsupport.org.au

March 2024 March 19th, 2024 – Ionis has just announced the opening of two enrolment locations in Japan – one in Tokyo and the other in Yamaguchi for the PrProfile Study. You can learn more about t...

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Video Presentation from 11th Annual National CJD Conference

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