Image includes a purple rectangle in the background of top third. The Chelsea's Hope Lafora Children Research Fund research roundtable logo is towards the left of the rectangle in white. It has half a bullseye to the left of the text. To the right of the logo's text is a purple line. On the other side, white text says 'Thursday, 8 February 10:30 AM - 12 PM EST.' a darket purple line separates the purple background from the white hexagons that make up the rest of the image. Below that, white text on a purple oval says 'REGISTER IN ADVANCE.' The photo on the left is a headshot of Sharmistha Mitra, Ph.D. with text beneath her name that says 'UT Southwestern Medical Center.' The photo on the right is a headshot of Jeff Milton with text beneath his name that says 'La Jolla Labs, Inc.'

Announcing February Research Roundtable Speakers

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Dr. Mitra and Jeff Milton to Join Chelsea’s Hope Lafora Disease Research Roundtable on 8 February

Image includes two round photos, with the Chelsea's Hope Lafora Children Research Fund research roundtable logo in the center. It has half a bullseye to the left of the text. A line is beneath. Text says 'Thursday, 8 February 10:30 AM - 12 PM EST' beneath the line. Below that, white text on a purple oval says 'REGISTER IN ADVANCE.' The photo on the left is a headshot of Sharmistha Mitra, Ph.D. with text beneath her name that says 'UT Southwestern Medical Center.' The photo on the right is a headshot of Jeff Milton with text beneath his name that says 'La Jolla Labs, Inc.' The background of the image is white hexagons.

We are excited to announce the two speakers that will present at our February research roundtable.

Sharmistha Mitra, Ph.D., is an Instructor in the Department of Pediatrics, Division of Child Neurology at UT Southwestern Medical Center. She joined the UT Southwestern faculty in July 2020.

Dr. Mitra’s objective is to present a comparative murine study of the amylopectinoses of APBD, Lafora Disease, and PGBM1 at the roundtable. She will share a mechanistic understanding of the function of E3 ubiquitin ligases and their associated proteins in glycogen metabolism.

Jeff Milton is the Founder and CEO of La Jolla Labs, a company developing technology for RNA therapeutics. Prior to this, he was Head of Data Sciences at Arcturus Therapeutics, where he worked on several rare disease programs, including both RNA-targeting and mRNA modalities. Jeff currently serves as an advisor to his alma mater, the Mellon College of Science at Carnegie Mellon University.

We are excited to begin the 2024 Research Roundtable series and hope you will join us for an important conversation.

Our Next Research Roundtable

Please join us Thursday, February 8th, from 10:30 am – 12:00 pm EST to learn from Jeff and Dr. Mitra. You must register for the Zoom meeting in advance. We will include a new translation and captions feature for the Zoom meeting. It will not be recorded, although we will share a summary after the meeting.

REGISTER NOW

 

Zaina headshot

Zaina Zaid Nemer

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Zaina in a wheelchair surrounded by three adults standing and smiling at a hospital

Zaina at UT Southwestern, July 2025

Zaina, from Gaza City, is the seventh patient in the ION283 Safety Study.

She was diagnosed with the ultra-rare epilepsy at the age of 13. Before her symptoms progressed, she loved to play basketball and was her team’s captain.

A stellar student, she applied herself to physical, occupational, and speech therapy just as she had in school. Zaina relied on medication to manage her seizures, and she even returned to school to take some exams.

2024: Help Lafora Patient Zaina and Family Evacuate Gaza

Zaina sat facing away from the camera with bags in the background

Zaina, October 2023

Everything changed in October 2023 – surrounded by violence and destruction, the hospital where she received treatment was no longer operable. Her family evacuated repeatedly to escape the fighting. There were times she lost the ability to speak and move because she was in status epilepticus, which terrified her mother. Her family was desperate to access medication and get to safety.

“There is nothing in pharmacies…There is no clean place here. We eat with the lids of cans, and I am careful when using them so as not to harm her,” shared her mother.

Zaina’s uncle started a GoFundMe in January 2024 to help her.

2025: Hope in Dallas

Zaina (left) celebrates her 19th birthday with her dad and Emi (seated) and her parents.

Zaina is one of 10 eligible patients participating in the ION283 Safety Study. Earlier in 2025, she made it to Egypt and recently traveled to Dallas, Texas, where she received her first dose in July 2025. Accompanied by her dad, Zaina has even had the opportunity to connect with other Lafora patients. She recently celebrated her 19th birthday in Dallas. Zaina and her family are so thankful for this opportunity and the hope it has brought them.

 

Help Lafora Patient Zaina and Family Evacuate Gaza

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Zaina smiling in a wheelchair with bags in the background Lafora patient Zaina and her family want to evacuate Gaza; donate to their GoFundMe today.

A 17-year-old from Gaza City has Lafora Disease.

Zaina sat facing away from the camera with bags in the backgroundHer entire family has been bombed, and they evacuated their home. They have moved across Gaza several times to escape the fighting. They have been displaced into a shelter and ran out of the best seizure medication for Zaina. Her family was able to switch epilepsy medications, but it’s not as effective. She needs proper medical treatment and a peaceful home.

Zaina’s mom shared some of their life, messaging, “There is nothing in pharmacies…There is no clean place here. We eat with the lids of cans, and I am careful when using them so as not to harm her. Everything is bad…”

Zaina’s uncle started a GoFundMe to help evacuate his family. You can give directly his efforts to get them through the Rafah crossing.

Yes, I want to help.

The video below shows Zaina last week. Her Lafora symptoms are progressing

Giving Tuesday November 28 Donate. Fundraise. Be generous.

2023 Annual Letter

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Below is Chelsea’s Hope annual progress update and appeal from our President, Frank Harris, Ph.D. If you did not receive a copy of the 2023 annual letter by email or direct mail, please email us. Then, we will get you set up for the future.

Read the Letter

Dear community, 

As we approach the end of another transformative year, we want to express our heartfelt gratitude for your unwavering support of Chelsea’s Hope. We have made significant strides toward our shared vision of a world with a cure for Lafora Disease. 

We are proud to share the progress we’ve achieved together in 2023. Your generosity has enabled us to expand our research network around the globe. Through the efforts of Chelsea’s Hope, Lafora Disease has been formally recognized by the International Classification of Diseases with an ICD-10 code. Chelsea’s Hope was awarded a grant in partnership with researchers at the University of Texas, Southwestern, and we co-hosted the 2023 Science Symposium. Chelsea’s Hope also started a Research Roundtables series, volunteer program, and sibling support program, all aligned with our mission of improving the lives of those affected by Lafora Disease.

The impact of your support is tangible: you have led us here. Your belief in our mission to help accelerate the development of treatments has fueled our determination despite the obstacles we’ve faced. There is still more we can achieve. 

We kindly ask for your continued support as we close out 2023. This year, Chelsea’s Hope is raising $20,000 by January 1, 2024, so we can continue expanding and maintaining a strong, collaborative, and actionable research network. By doing so, we will be closer to starting a clinical trial. 

Lafora Disease progresses quickly, and time is never on our side. We hope you will be. 

Your donation is more than a financial contribution. It is an investment in saving the lives of the hundreds of children and their families fighting this terrible disease.

We are so grateful to be able to count on you to help our children struggling today and to honor the legacy of those we lost. Thank you for being an essential part of our work. You are building a better future for all affected by Lafora Disease.

I wish you and your loved ones a joyous holiday season and a happy new year.

With Hope, 

 

 

 

Frank Harris, Ph.D. – President

Chelsea’s Hope Lafora Children Research Fund

Post Office Box 348626, Sacramento, CA 95834

info@chelseashope.org

Reach a Cure for Lafora Disease

Your support allows us to continue the vital work we do to reach a cure. This year-end, you can create your own fundraiser to help us reach our goal. Double your impact when you donate, because a generous community leader has offered a $10,000 match for every gift made before December 31, 2023.

Donate here