Tag Archive for: ion283 safety study

18 June 2025 wednesday Register online to attend the Zoom meeting. ION283: what's next? Q&A Submit your questions beforehand.

Announcing June 18th ION283 Q&A: What’s Next?

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Questions about the ION283 Safety Study or what’s next?  We hope you will join us for a Zoom ION283 Q&A next Wednesday, June 18th, at 4 p.m. ET.

Please register in advance to attend the meeting.

While we have our FAQs, a lot has changed over the past year. We are so thankful that members of the UTSW team will join us at the Q&A to share what they know. Please submit questions through this form by Monday, June 16th, for Dr. Minassian and Dr. Messahel to review them before the meeting.

Chart showing 6/10 patients enrolled in the ION283 safety study and 7/10 patients funded

Six patients have been enrolled in the ION283 Safety Study. We have funded the seventh participant and need to raise more for the final three children to join.

We will use Zoom’s closed caption translation feature. Please do not hesitate to join us in the ION283 Q&A or submit a question for us to ask if your first language is not English.

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Chelsea’s Hope Advisory Board Meets to Discuss Research Goals

Chelsea’s Hope Advisory Board Meets to Discuss Research Goals

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The Chelsea’s Hope Advisory Board met in April to provide guidance and support for the organization’s clinical and research goals in 2025:

  1. Get the ION283 safety study fully funded
  2. Assist with the analysis and publication of collected data in order to drive biomarker development for Lafora disease and determine global prevalence rates
  3. Establish Clinical Centers of Excellence and/or Guidelines for Clinicians

The primary topics of discussion were preparing for ION283 clinical trials, validating biomarkers for Lafora disease progression, and developing guidelines for seizure management for Lafora patients. 

ION283 Safety Study

The ION283 Safety Study now has six patients enrolled. Dr. Berge Minassian, who directs the study at the University of Texas Southwestern Medical Center, assured the board that patients will be enrolled as soon as funds are available. Also, Noventia is continuing its preparations to launch a clinical trial for ION283 in Europe. The timeline and details of this trial are still under discussion. Members of the Advisory Board will make themselves available to Noventia when requested to provide expertise and insight to ensure the best care for Lafora patients.

Four patients wait to join the ION283 Safety Study.

Biomarker Research Goals

Before any potential Lafora therapy can begin clinical trials to establish efficacy, biomarkers for Lafora disease progression must be identified and validated. Chelsea’s Hope and the broader Lafora community have assisted with global efforts to collect patient samples for biomarker analysis. The data from the two-year Natural History Study sponsored by Ionis is now ready for analysis. Members of the Advisory Board have committed to participate in working groups over the summer to review the data and recommend promising biomarkers.

Centers of Excellence

While our organization continues to support the development of therapies, there are steps that we can take now to improve the quality of patient care worldwide. This year, Chelsea’s Hope will sponsor a series of discussion panels to establish Centers of Excellence for Lafora Disease and guidelines for patient care. The Advisory Board recommended that the organization prioritize the development of guidelines for seizure management in Lafora disease. Dr. Roberto Michelucci will direct this effort with support from Chelsea’s Hope as well as a cohort of clinicians who treat Lafora patients.

Questions?

For more information about the Chelsea’s Hope Advisory Board,the projects, or the research goals outlined in this news post, please contact our Scientific and Executive Director, Dr. Kit Donohue (katherine@chelseashope.org). 

Town hall To discuss fundraising efforts and plan as a community. FULLY FUND THE SAFETY STUDY21 MAY 2025 WEDNESDAY Southwestern William P Clements Jr. U Register online to attend the Zoom meeting.

Announcing a Town Hall: Fully Funding the Safety Study

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We hope you will join us for a Zoom Town Hall next Wednesday, May 21st, at 4 p.m. ET, to discuss fully funding the safety study. Register to attend today!

We have less than a month to fundraise to enroll the final four patients on the original timeline. Learn more about our fundraising initiatives and how you can get involved when you join our Town Hall next week.

Six out of 10 patients have been enrolled in the ION283 Safety Study.

We will use Zoom’s closed caption translation feature. Please do not hesitate to join us if your first language is not English.

Wondering about what’s next for ION283 development? Check our ION283 Safety Study FAQs and stay tuned for more information about a Q&A in June. We will not discuss that next week; we will focus on fundraising planning.

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Chelsea's Hope Lafora Children Research Fund's 2024 Annual Report

Read 2024 Impact in our Annual Report

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Chelsea’s Hope recently published its 2024 Annual Report. Inside, you’ll find a letter from our President, a breakdown of our 2024 impact, financials, and what we’re working towards in 2025.

To our generous donors, dedicated research network, and community members, this is your impact, too. Every new resource or hopeful advancement to treatments is thanks to your support!

We often say that together, we fight Lafora disease. That is true more than ever as we are united as a worldwide Lafora community to support the ION283 safety study.

“While we celebrated this milestone, we acknowledge that many families are still without a path to treatment for their loved ones. Every child matters. We will continue to support all of our Lafora families and push for expanded access to treatments and new therapy development.” – Jenifer Merriam, President

Much of the work you’ll read about in the Annual Report was possible thanks to grants from the Chan Zuckerberg Initiative. We also received event support from the Danny Did Foundation, Vibe Bio, Tempo Zero, A.I.L.A., France Lafora, and Association Cel-luz. We appreciate all our 2024 supporters and your continued partnership to reach a cure!

2025 Goals

Last year, despite progress, also left us mourning the passing of more children. We will keep fighting for a better future in their memory.

Our goals for 2025 are to: 

  • Get the ION283 safety study fully-funded (we are more than halfway there)
  • Establish Clinical Centers of Excellence & Guidelines for Clinicians
  • Publish collected data in order to:
    • Drive biomarker development
    • Determine Lafora prevalence rates
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Are you interested in getting involved? Stay connected by signing up for our mailing list and following our social media for regular updates. We always welcome donations and volunteers to support our cause.

We remain committed to improving the lives of those affected by Lafora and helping accelerate the development of treatments. Thank you for your belief in our mission!

Download the 2024 Annual Report