Lafora Disease Centers of Excellence Listening Sessions

Invitation to Join Listening Sessions

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Families and caregivers, you might have seen in our July newsletter that we need your help to establish criteria for Centers of Excellence for treating Lafora disease.

As an ultra-rare disease, most hospitals do not know what resources are needed in order to provide the best possible care for our children with Lafora. By establishing guidelines for Centers of Excellence, we can provide a set of recommendations for treating Lafora patients and recognize hospitals that provide exemplary care for our children with Lafora.

We invite you to join us at one of the listening sessions in August to share your advice on what support you receive (or need) from your care facility. There are currently four sessions scheduled; please sign up for the session with the time and language that works best for you.

Listening Sessions

August 1: English (but all languages welcome)
Join Jenifer at 10 AM Pacific Daylight Time. >

August 8: Hindi/English
Join Vaishali at 7:30 PM India Standard Time. >

August 15: Spanish
Join Nancy at 10 AM Eastern Daylight Time. >

August 25: Italian
Join Giuseppe at 7 PM Central European Summer Time. >

Together, we can advocate for better care. By recognizing locations with expertise, we can learn from their practices and improve worldwide treatment options for Lafora patients.

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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
Noventia Pharma logoNoventia’s Reply to Lafora Patient Community