Tag Archive for: 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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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
ION238 Safety Study Update February 2025

Safety Study Update – February 2025

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We are excited to share an update about the ION283 Safety Study: the team at UTSW can continue enrolling patients. 

In December 2024, the first patient in the Lafora Disease Safety Study at the University of Texas Southwestern Medical Center (USTW) received their first dose of ION283, a treatment designed to halt disease progression. Last month, that patient completed their one-month check-up with no safety issues detected. This allowed a second patient to receive their first dose of ION283 in January. Earlier in February, the second patient completed their one-month safety check. The safety board at the university reviewed the safety data, giving an all clear to proceed as planned with the study.

The first ten families who applied to participate in the study will receive notification from the team at UTSW when there is enough money available to enroll them in the study. We are pleased to share that the third patient is en route to Dallas, and the fourth has been notified.

As a community, we must continue raising funds so all 10 patients can receive treatment. The data collected from this study will help accelerate the approval process so that we can expand access for other patients to receive this drug. However, we need to complete the enrollment for this study first and get the data we need. So far, we have raised more than $977,000 as a community. We must reach 1.5 million dollars by May 2025 to fully fund the study. 

We will continue to keep the Lafora community informed about the progression of the Safety Study. Any questions not answered in our FAQs about the study can be directed to Chelsea’s Hope at info@chelseashope.org.

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