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Attendees at the Lafora disease science Symposium 2023 in Bologna

Lafora Symposium 2023 in review

October 11, 2023/by Christine Kelly

Attendees at the Lafora disease science Symposium 2023 in Bologna

The 8th Lafora Disease Science Symposium was held October 9-10th, 2023, in Bologna, Italy. It was the largest event yet, with more than 30 researchers, clinicians, and patient advocates gathered to share their expertise on Lafora Disease. The community joined worldwide to work toward improving treatment for Lafora patients.

Thank you to all the attendees

The team at Chelsea’s Hope was grateful to be able to meet in person and connect with our incredible partners and families who support our mission.

Families, you are why we exist. Reaching a cure for our children motivates our work daily, and we know you share that goal. We are thankful to be a part of a committed community worldwide that is fighting Lafora Disease.

To all speakers, thank you for sharing your time and expertise with the Lafora Community at our Symposium this week! Your presentations gave us hope that better treatment options for our patients are coming soon. Truly, we are so grateful for the Lafora research network and dedicated clinicians who collaborate to find a cure for our kids. 

Thank you to the fantastic hosts in Bologna from ISNB IRCCS. You made us all feel welcome, and we look forward to future symposiums in Italy.

We are immensely grateful to our partner organizations, whom we commit to continue connecting with to advance our joint fight against Lafora Disease.

Finally, to all attendees of the 2023 Lafora Symposium, thank you for joining us in Bologna! If you have any photos from the Symposium that you’d like to share, please upload them HERE. Anyone can view the photos there by clicking the link. With a Google Drive account, anyone can upload pictures, too. Use #LaforaSymposium23 on social media and tag us @chelseashopelaforacure!

Connecting with other families and hearing new data, research, and ideas from the Symposium speakers inspired a new level of hope in us as an organization. A key takeaway for us is that together, we can achieve more.

Together, we can fight Lafora.

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https://chelseashope.org/wp-content/uploads/2023/10/Symposium-2023-Attendees-scaled.jpeg 1168 2560 Christine Kelly https://chelseashope.org/wp-content/uploads/2022/02/ChelseasHope1.png Christine Kelly2023-10-11 09:05:592024-11-30 07:14:00Lafora Symposium 2023 in review
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Lafora Therapy Development Pathway This pathway does not guarantee therapy approval, but instead shows the necessary steps a therapy must go through to become available to patients. Discovery & Early Development Early Preclinical Testing Advanced Preclinical Steps Human Safety Study & Dose Escalation Efficacy Study Regulatory Review Continued Monitoring Therapies are designed, synthesized, and evaluated for purity and stability. Therapies are tested in cells or mouse models to demonstrate safety and efficacy. Therapies are tested in animal models to validate the delivery method, dosing levels and dosing frequency A Phase I clinical study focuses on demonstrating safety in patients and finding an effective dosage. A Phase II/Ill study focuses on proving the therapy meaningfully slows or halts disease progression Review of data from the clinical trials by agencies such as the FDA are required before therapy approval. After approval, therapies may continue to be monitored for safety, effectiveness, and long-term outcomes. VAL-1221 Brain delivery is a major limitation, and therapy is currently not moving forward as an active Lafora disease therapy. Next-Generation Enzyme Fusion Therapies Preclinical work is focused on improving delivery to the brain using IV administration. EPM2A/EPM2B Gene Therapy Preclinical research to replace the mutated genes causing Lafora disease. Currently needing to complete toxicology (safety) studies Amylase Gene Therapy Preclinical work in progress to confirm amylase expression in cells and the ability to clear Lafora bodies (efficacy). VAL-0417 A preclinical enzyme-fusion therapy designed to help clear existing Lafora bodies. ICV delivery is being tested to confirm the drug reaches the brain before it can advance to the clinic. ION283 The current safety study has 10 patients who have received low levels the drug with no major safety issues. They are now in the dose escalation phase determine an effective dose.Chelsea's Hope Lafora Children Research Fund 2026 Lafora Therapy Q&A Resources

About Chelsea’s Hope

Chelsea’s Hope began as a website in the fall of 2007 to share Chelsea Gerber’s story with family and friends. A 501(c)(3) organization since 2009, we have made an impact by raising awareness, connecting families worldwide, funding research, and maintaining hope.

Our mission is to improve the lives of those affected by Lafora disease and help accelerate the development of treatments. [Read More]

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The mission of Chelsea’s Hope is to improve the lives of those affected by Lafora disease and help accelerate the development of treatments.

Chelsea’s Hope Lafora Children Research Fund is an IRS 501(c)3 nonprofit organization. EIN: 27-1008382

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Use ICD-10 code G40.C for Lafora.

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