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A SABATO!
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Each year the Family Conference gathers researchers, medical professionals and families in person. Here are some of the highlights.
Family Workshops: Friday focuses on families, with sessions including Sporadic CJD Workshop, Genetic Prion Disease Workshop, Clinical Prion Disease Basics Information Session, Prion Disease Research Overview, and Bereavement Workshop. The day concludes with a welcome reception to families to meet one another or catch up with friends met in prior years.
Keynote Presentation: In 2024, Simon Mead, BM BCh, FRCP, PhD, Clinical Professor, MRC Prion Unit at UC London, Institute of Prion Diseases, London, England, presented the Keynote Address in Honor of Dr. Pierluigi Gambetti: Can Genetics Help in the Fight Against CJD?
Research Grant Reports: Thanks to our supporters, the CJD Foundation funds many research grants each year, and each of those researchers provided an overview of their work at the conference, followed by Q&A sessions.
Moving Roundtables: In this segment, always a favorite of both families and professionals, families rotate among tables in small groups, and sit for informal Q&A with keynote and other speakers, workshop leaders, and grant recipients.
CDC and NPDPSC Updates: Experts from the Centers for Disease Control and Prevention and National Prion Disease Pathology Surveillance Center provide updates on their important work.
Family Panel: Hear directly from family members whose loved ones had unique experiences with CJD and learned how they are now making a difference in the fight against CJD.
Banquet: At each year’s banquet, we present an overview of the CJD Foundation’s accomplishments and year in review. We are also pleased to recognize some key volunteers and leaders in the prion disease community. Winners of the Strides for CJD 2024 fundraising competition will also be recognized and memorial tributes to their loved ones and other highlights are shared.
DCJBrazil Family Panel “A global union for prion disease awareness and support”
Description: This family panel brings together voices from across the prion disease community to share their lived experiences. Through personal stories, challenges, and moments of hope, families offer unique insight into life with a rare disease. This panel will also include a speech about how families can handle this very difficult time and an enlightening talk about prion disease therapies.
Format and duration: This panel will be in a hybrid format and will especially happen on November 5th.
Target audience: Families affected by prion diseases.
Relevance: The aims of this panel are:
• Empower families by sharing their journeys
• Encourage community support and advocacy
• Help families how to handle this process
• Inform about prion disease therapies
Restrictions on participation: Participation by invitation. For invitations, please contact Eriton Cunha via email (prionsprotein@gmail.com).
December 30th, 2024 – Enrolment is now complete for the Ionis Phase 1/2a clinical trial of ION717 called PrProfile for symptomatic prion disease patients. Updates will be provided as they become available. Please review the attached Community Statement.
August 16th, 2024 -We are pleased to share with you the attached Community Statement from Ionis announcing that the pause on recruitment for their PrProfile Prion Disease study has been lifted. In addition, all planned clinical trial locations for PrProfile are now active, including several additional clinical trial sites that were not part of the earlier stage of enrollment – 16 in total.
Please review the attached Community Statement. We remain hopeful with the continued progress toward potential drug developments and clinical trials that may lead to a treatment or cure.
April 9th, 2024 -Ionis has released the following community statement on the PrProfile clinical trial recruitment pause:
March 26th, 2024 – Ionis has just announced the activation of a site in Gunma, Japan for the PrProfile Study.
You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
March 25th, 2024 – Ionis has just announced the activation of a site in Tel Aviv, Israel for the PrProfile Study.
You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
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 this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
March 13th, 2024 – Ionis has just announced the opening of an enrolment site in Montreal, Canada (McGill University Health Centre) for the PrProfile Study.
You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
February 21st, 2024 – Ionis has just announced the opening of an enrolment site in Boston, USA (Massachusetts General Hospital) for the PrProfile Study.
You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
February 3rd, 2024 – Ionis has just announced the opening of an enrolment site in Germany for the PrProfile Study. This is the third site to open outside of the USA.
You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
January 27th, 2024 – Ionis has just announced the opening of an enrolment site in Barcelona, Spain for the PrProfile Study. This is the second site to open outside of the USA.
You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
January 24th, 2024 – Ionis has announced that they have opened a third enrollment site in Canada (University of Alberta) for the PrProfile Study.
You can learn more about that site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.
Anyone interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.
As of January 20, 2024, the UCLA Neurology Clinic in Los Angeles, California, United States has been added as the next prion disease human clinical trial site.
Please email PrionDisease@clinicaltrialmedia.com or call (844) 221-3587 to obtain more information on the study details and clinical trial site locations.
The status of the Ionis Clinical Trial on clinicaltrials.gov is now “Recruiting“.
One site is listed as active and recruiting at this time and that is in the U.S. at the University Hospitals Cleveland Medical Center in Ohio.
Key points:
We encourage you to:
Remember, this is just a first step in a very long process. Research does take time but each step leads us closer to discovery. Regardless of the outcome, this is a positive step for those impacted by prion disease.
The Ionis Clinical Trial is posted on the clinical trials website.
Here is the link: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
The current status is “not yet recruiting”. Even though no sites are open, this is a normal step in the process. You are able to see some of the study criteria and other information.
We will continue to provide you with updates as things progress.
Post and share that you are ‘Globally united for prion disease awareness‘ to your local CJD/prion disease community to raise awareness.
Available to download are:
International CJD Awareness Day – A4 poster (pdf)
Prion disease infographic (pdf)
Social media graphics: