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ION238 Safety Study Update February 2025

Safety Study Update – February 2025

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.

 

This is the Platinum Seal of Transparency from Candid. It has a platinum border, white square center, and text that says 'Platinum Transparency 2025 Candid.'

Chelsea’s Hope Earns Platinum Seal of Transparency for 2025

This is the Platinum Seal of Transparency from Candid. It has a platinum border, white square center, and text that says 'Platinum Transparency 2025 Candid.'Chelsea’s Hope is excited to announce we’ve just earned a Platinum Seal of Transparency from Candid for 2025!

We invite you to check out our nonprofit’s work through our Candid nonprofit profile; where you can view our impact.

We rely on our profile to gather support; it enables funders and donors to find us and learn about our impact. We’ll keep our profile updated so you can see what your donations help us accomplish. Recently, we shared information about our mission, programs, results, financials, and operations.

Chelsea’s Hope wants to ensure you have the progress updates you need to support our mission with trust and confidence. Your contributions make a real difference. You can donate knowing our commitment to operating with the highest level of transparency, as evidenced by our achievement of the Platinum Seal for 2025.

What’s next? 

In 2025, we plan to host three more Research Roundtables and support the Lafora Disease Science Symposium. We must fully fund the safety study to enroll all the eligible patients, too. As always, we will champion the amazing Lafora community and take every step forward to treatments for our children. Finally, you can look forward to monthly family support groups.

Thank you for your belief in our cause!

20252-2026 Welcoming a New Board of Directors Katrina Voe Cotton Vice President Roderick Howard At-Large Member

Chelsea’s Hope Welcomes 2025-2026 Board of Directors

Chelsea’s Hope is pleased to announce the 2025-2026 Board of Directors: Jenifer Merriam (President), Dr. Katrina Voe Cotton (Vice President), Sheila Barter (Treasurer), Meredith Williams (Secretary), Niki Markou (At-Large Member), Roderick Howard (At-Large Member), and Nadia Ismail (At-Large Member).

New to Chelsea’s Hope are Vice President Katrina Voe Cotton, MD, PhD, and At-Large Board Member Roderick Howard.

Headshot of Katrina Voe Cotton, MD PhD

Dr. Katrina Voe Cotton, Vice President

Dr. Cotton shared, “I feel incredibly fortunate to have joined the Lafora community at such an exciting time. With the safety study now underway at UT Southwestern, we are on the brink of a new era for Lafora patients. I’m eager to leverage my experience and knowledge to help navigate this exciting journey ahead.”

Headshot of Roderick Howard

Roderick Howard, At-Large Member

Roderick, a longtime nonprofit leader, also said, “I am grateful for the opportunity to serve an organization that aligns with my interests in public health and children’s support.”

Dr. Cotton and Roderick bring a wealth of management and development skills along with a passion for making a difference in the Lafora community. We look forward to their guidance and expertise in advancing the mission of Chelsea’s Hope. You can read more about the Board of Directors on our Leadership Team page

Finally, we want to express our heartfelt gratitude to Dr. Frank Harris for his leadership as the previous President of the Board of Directors. He had a talent for making newly diagnosed families feel supported and devoted a decade to serving the Lafora community with Chelsea’s Hope. Many of our current board members credit Frank for welcoming them to the organization and encouraging them to join. While he stepped down from the board, he remains a steadfast member of our community.

In Memory of Dr. Joan Guinovart

Headshot of Dr. Joan J. GuinovartWe are deeply saddened by the loss on January 1st, 2025, of Professor Dr. Joan Guinovart — founder, former director, former researcher, and professor of IRB Barcelona, an international research center devoted to curing cancer and other diseases linked to aging. He was a brilliant scientist and a wonderful human being. Dr. Guinovart’s research was primarily concerned with glycogen, starting with a study of the effects of insulin on glycogen synthase and eventually leading to the study of Lafora disease, a debilitating disease characterized by the accumulation of abnormal glycogen. His research was instrumental in identifying a therapeutic target to treat patients with Lafora disease.

The team at Chelsea’s Hope is extremely thankful for Dr. Guinovart’s research and the milestones he created to get us to treatments. He will be missed by many in our Lafora community. We are sending our condolences to his family and friends.