Dear Noventia Pharma Team, We are families affected by Lafora disease who are holding on to hope through your work. Your dedication to developing a treatment for Lafora means everything to us. We were excited when you shared the news of the ION283 license purchase and your intentions to create a clinical trial last year, but we are anxiously awaiting more details. We know that any future progress with ION283 will come from your company, so we kindly request a public update on what is next for the drug’s development after the safety study at UTSW. As you know, the current ION283 safety study includes only 10 children. While we are grateful this study is happening, many more families were heartbroken to learn our children could not participate. These families are now left waiting, watching their children’s condition progress, and praying for the next opportunity. We are counting on Noventia to help make that opportunity possible. For many of us, your work is the only hope we have left. Our children do not have time to wait. Lafora disease is aggressive and unforgiving—we are pleading for a chance to save our children’s lives. We desperately urge Noventia to share your plans and move forward quickly with a clinical trial or expanded access program so more children can receive this potentially life-saving treatment. You have our full support in this mission, and we are willing to assist however we can.

Open Letter to Noventia

Posted: July 3, 2025

 

A graphic with nine headshots introducing new team members; text says: Meet the team sophie stein research support intern jeremiah paul fundraising support intern samy sharif science communications intern sara gerber research support intern sally leung research support intern jhanavi kotian science communications intern kait fedor development fellow vaishali jain fundraising support intern anna gould science communications intern

New Team Members Join Chelsea’s Hope for the Summer

Posted: June 24, 2025

We are thrilled to introduce nine new team members who will be joining us this summer. Chelsea’s Hope was amazed when we had more than 500 applications for our 2025 internship program. As the organization seeks to expand its capabilities to achieve our mission, we have brought on as many team members as possible to […]

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?

Posted: June 10, 2025

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 […]

Chelsea’s Hope Advisory Board Meets to Discuss Research Goals

Chelsea’s Hope Advisory Board Meets to Discuss Research Goals

Posted: May 19, 2025

The Chelsea’s Hope Advisory Board met in April to provide guidance and support for the organization’s clinical and research goals in 2025: Get the ION283 safety study fully funded Assist with the analysis and publication of collected data in order to drive biomarker development for Lafora disease and determine global prevalence rates Establish Clinical Centers […]